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MG News Coverage and MGFA Press Releases
The latest info for the MG community is right here!
After successfully completing phase 1 single ascending dose in healthy volunteers, NMD670 was tested in patients with MG in a randomized, placebo-controlled, single-dose, three-way crossover clinical trial. The clinical trial evaluated safety, pharmacokinetics, and pharmacodynamics of NMD670 in 12 patients with mild MG. NMD670 had a favorable safety profile and led to clinically relevant improvements in the quantitative myasthenia gravis (QMG) total score. This translational study spanning from single muscle fiber recordings to patients provides proof of mechanism for ClC-1 inhibition as a potential therapeutic approach in MG and supports further development of NMD670.
View the published data: https://www.science.org/doi/10.1126/scitranslmed.adk9109?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed
NMD has opened phase 2 and is currently active and recruiting MG patients. See the following ClinicalTrials.gov page to determine your eligibility and where to apply to be part of this exciting trial. https://clinicaltrials.gov/study/NCT06414954?term=NMD670&rank=2
MGFA Awards $330,000 in Research Grant Funding
MGFA News Release
September 6, 2024
Myasthenia Gravis Foundation of America is pleased to announce our latest grant recipients. Three researchers were selected for the 2024 MGFA High Impact Pilot Project Award. Their work represents an exciting step forward in our understanding of myasthenia gravis.
Learn more about the recipients and their research
Does Surgical Removal of the Thymus Have Deleterious Consequences?
Neurology
May 24, 2024
Recently, a concern has been raised about deleterious consequences of the surgical removal of thymic tissue, including for patients who undergo thymectomy for myasthenia gravis (MG) or resection of a thymoma. This review adopts a multidisciplinary approach to scrutinize the evidence concerning the long-term risks of cancer and autoimmunity postthymectomy. We conclude that for patients with acetylcholine receptor antibody-positive MG
and those diagnosed with thymoma, the removal of the thymus offers prominent benefits that well outweigh the potential risks. However, incidental removal of thymic tissue during other thoracic surgeries should be minimized whenever feasible.
Comparative effectiveness of azathioprine and mycophenolate mofetil for myasthenia gravis (PROMISE-MG): a prospective cohort study
The Lancet Neurology
March 2024
This study aimed to evaluate the comparative effectiveness of azathioprine and mycophenolate mofetil in MG treatment, and to assess the effect of the dose and duration of treatment. In the study, more than half of patients treated with azathioprine and mycophenolate mofetil felt their quality of life improved; no difference in clinical outcomes was noted between the two drugs. Adverse events associated with azathioprine were potentially more serious than those with mycophenolate mofetil, although mycophenolate mofetil is teratogenic. Lower-than-recommended doses of azathioprine might be effective, with reduced dose-dependent adverse events. More comparative effectiveness studies are required to inform treatment choices in myasthenia gravis.
This study was funded, in part, by the Myasthenia Gravis Foundation of America.
Myasthenia Gravis Foundation of America (MGFA) Kicks off Annual National MG Patient Conference With Program Announcements
MGFA News Release
April 27, 2024
More than 400 members of the myasthenia gravis (MG) rare disease community will come together this week at the MGFA National Patient Conference for support and a better understanding of how to manage their disease while learning about the latest in treatments and clinical studies. MGFA is making a series of announcements at the event while highlighting new information and programs to help individuals living with MG.
Fifty Plus Advocate
February 2024
Glenda Thomas, MGFA support group leader, has been sharing her story for several years, including in a recent article in a local paper – check it out on page 16. She also discusses the importance of support groups when you are navigating a chronic illness like myasthenia gravis.
MGFA Medical and Scientific Advisors’ Statement on Recent New Candidate Biomarker Research Report
February 8, 2024
You may have read recently that a University of Alberta research team announced that it may have identified a candidate universal biomarker (fibrinogen – a key component of blood clotting) for myasthenia gravis (MG). In newly published research, the team reports that it used advanced proteomics techniques to identify this candidate biomarker that can be detected with a blood test to provide an MG diagnosis.
The medical and scientific advisors at the Myasthenia Gravis Foundation of America (MGFA) have evaluated this report and agree that the findings are intriguing and may have considerable impact in both MG research and clinical care. That said, as with all new scientific discoveries, independent validation will be required to confirm the findings. We look forward to additional research in this area to understand the possible role and utility of fibrinogen in MG. Biomarker research remains a critical priority in the field of autoimmune MG.
ZILBRYSQ® (zilucoplan) Is Now Commercially Available in the U.S. for the Treatment of Generalized Myasthenia Gravis (gMG) in Adult Patients Who Are Anti-Acetylcholine Receptor (AChR) Antibody Positive
UCB Website
January 3, 2024
UCB, a global biopharmaceutical company, announced today that ZILBRYSQ® (zilucoplan) is now available in the U.S. for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. ZILBRYSQ was approved by the U.S. Food and Drug Administration (FDA) on October 17, 2023.1
ZILBRYSQ is available by prescription as a ready-to-use pre-filled syringe that is a once-daily administration.
UCB Announces U.S. FDA Approval of ZILBRYSQ[®] (zilucoplan) for the Subcutaneous Treatment of Adults with Generalized Myasthenia Gravis
UCB Website
October 17, 2023
UCB announced that ZILBRYSQ® (zilucoplan) has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody-positive. Zilucoplan is the first once-daily subcutaneous, targeted C5 complement inhibitor for gMG and is self-administered.
Zilucoplan is the first once-daily subcutaneous (SC), targeted peptide inhibitor of complement component 5 (C5 inhibitor). It is the only once-daily gMG target therapy for self-administration by adult patients with anti-AChR antibody-positive gMG. Benefits of self-administered treatment can include reduced traveling time to and from hospitals, decreased interference with work obligations, and increased independence. Unlike monoclonal antibody C5 inhibitors, as a peptide, zilucoplan can be used with intravenous immunoglobulin and plasma exchange, without the need for supplemental dosing.
The FDA approval of zilucoplan1 is supported by safety and efficacy data from the RAISE study (NCT04115293), published in The Lancet Neurology in May 2023. The RAISE study was a multi-center, phase 3, randomized, double-blind, placebo-controlled study to assess the efficacy, safety profile, and tolerability of zilucoplan in adult patients with anti-acetylcholine receptor (AChR) antibody-positive gMG.
“This is an important development for the community because, with more FDA-approved treatments for generalized myasthenia gravis, physicians have additional tools to treat this disease in individualized ways that are the right fit for each individual patient,” said Samantha Masterson, President and Chief Executive Officer of the Myasthenia Gravis Foundation of America. “We are so grateful to UCB for being part of the myasthenia gravis community and their continued commitment to finding solutions for people living with this chronic, autoimmune, neuromuscular disease.”
With the approval of zilucoplan, alongside the company’s neonatal Fc receptor (FcRn) blocker RYSTIGGO® (rozanolixizumab-noli), which was approved earlier this year by the FDA, UCB’s portfolio provides healthcare professionals the option of addressing either complement activation or pathogenic auto-antibodies for appropriate patients.
Read the Official News Announcement from UCB.
Assessing High-dose Chemotherapy and Hematopoietic Cell Transplantation in Severe Myasthenia Gravis
Annals of Clinical and Translational Neurology
September 19, 2023
Recently published report: Remission of severe myasthenia gravis after autologous stem cell transplantation
Summary by: Dakota Campbell –Communications Program Manager- Rare Diseases Clinical Research Network, Cincinnati Children’s, Cincinnati, OH 45229
Myasthenia gravis (MG) is a rare neuromuscular disorder caused by an autoimmune response which blocks or damages acetylcholine receptors on muscles. High-dose chemotherapy (HDIT) and autologous hematopoietic cell transplantation (HCT), also known as bone marrow transplant, are potential treatments for MG.
In this study, researchers investigated the safety and efficacy of HDIT and HCT in a patient with severe, treatment-resistant MG.
Results show that HDIT and HCT induced remission of MG. The team assessed the effect of treatment on the underlying immunopathology. Intriguingly, the acetylcholine receptor autoantibodies (AChR)—the known pathogenic mediators of the disease—did not appreciably lower after the treatment.
Authors state that these findings suggest a cell-based disease mechanism, which responds to high-dose therapy, may play a role in the pathology in addition to AChR autoantibodies. Further studies are needed to establish whether HDIT and HCT can be an effective therapy for severe MG.
You can read the entire paper in the Wiley.com library: https://onlinelibrary.wiley.com/doi/10.1002/acn3.51898
Telehealth in Myasthenia Gravis: What We’re Learning from a Pilot Study
Myasthenia Gravis Rare Disease Network
August 22, 2023
To learn more about the use of telehealth in MG, the Myasthenia Gravis Rare Disease Network (MGNet) is conducting a pilot study, “Adapting Disease Specific Outcome Measures Pilot Trial for Telehealth in Myasthenia Gravis (ADAPT-teleMG)” (clinicaltrials.gov ID NCT05917184). The team is evaluating telehealth visits and remote disease-specific assessments for patients with MG.
Here, lead investigator Amanda Guidon, MD, MPH, and Meridith O’Connor, MG patient and assistant vice president of patient engagement, advocacy, and policy at the Myasthenia Gravis Foundation of America (MGFA), share more about the study and its impact on the patient and research community.
Read More about this published research HERE
Modifying Patient T-cells Creates Novel Approach to Treating Myasthenia Gravis and other Diseases
Cartesian Website
July 1, 2023
In an effort to keep the MG Community updated on new research discoveries, we want to tell you about newly-published research in The Lancet Neurology.
Cartesian Therapeutics, an MGFA industry partner, announced the publication of positive results of the first successful clinical trial of RNA cell therapy for patients with autoimmune disease. By modifying patients’ T-cells with mRNA (a form of rCAR-T therapy), the study has created a novel approach for potentially treating myasthenia gravis (MG) and other autoimmune diseases.
The data demonstrates potent and durable clinical improvement in patients with MG, representing the first successful Phase 2 trial using RNA cell therapy.
“We are grateful to our community of MG patients and physicians for enabling clinical development of novel therapeutics such as rCAR-T,” said Samantha Masterson, President & CEO of Myasthenia Gravis Foundation of America. “A safe, personalized therapy with durable clinical benefit would be a welcome addition to the growing toolkit of MG treatments.”
The results described in The Lancet Neurology paper suggest that rCAR-T may be useful in treating a variety of other autoimmune diseases and may overcome many of the risks and toxicities associated with conventional DNA-based CAR-T cells. The news release “Safety and Efficacy of Autologous RNA Chimeric Antigen Receptor T-cell (rCAR-T) Therapy in Myasthenia Gravis: a prospective, multicenter, open-label, non-randomized phase 1b/2a study”, shares more details.
You can also download the full manuscript.
To learn more about Cartesian’s study with rCAR-T, or review other open and recruiting MG clinical trials that you can apply to, visit the MGFA Clinical Trials page.
UCB announces U.S. FDA approval of RYSTIGGO® for treatment of generalized myasthenia gravis
UCB Website
June 27, 2023
More amazing and groundbreaking news from our industry partners. UCB has just announced U.S. Food and Drug Administration (FDA) approval of a new treatment called RYSTIGGO (rozanolixizumab-noli) for adults with generalized myasthenia gravis who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.
Rozanolixizumab-noli is a subcutaneous infusion injection and the only FDA-approved treatment in adults for both anti-AChR AND anti-MuSK antibody-positive gMG, the two most common subtypes of gMG.
U.S. FDA approval is based on the pivotal Phase 3 MycarinG study in gMG2, a large phase 3 study which demonstrated treatment with rozanolixizumab-noli that resulted in statistically significant improvements in gMG-specific outcomes, including everyday activities such as breathing, talking, swallowing, and being able to rise from a chair.
“We want to thank UCB for their continued commitment to the MG community to bring a new FDA-approved treatment option for generalized myasthenia gravis to patients and their treating physicians,” said Samantha Masterson, President and Chief Executive Officer of the Myasthenia Gravis Foundation of America (MGFA). “People living with generalized myasthenia gravis continue to experience significant unmet medical needs, this means expanding the number of FDA-approved treatment options is particularly important to treat this chronic, autoimmune, neuromuscular disease.”
Rozanolixizumab-noli will be commercially available in the U.S. during the 3rd quarter of 2023.
Read the official news release from UCB.
argenx Announces U.S. FDA Approval of VYVGART Hytrulo Injection for Subcue Use in Generalized MG
argenx website
June 21, 2023
We are excited to inform you that another effective treatment for generalized myasthenia gravis has been approved by the U.S. Food & Drug Administration. argenx has announced that VYVGART® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc), a subcutaneous injection for adult MG patients who are anti-acetylcholine receptor (AChR) antibody positive.
According to our industry partner argenx, VYVGART Hytrulo is a subcutaneous product combination of efgartigimod alfa, a human IgG1 antibody fragment marketed for intravenous use as VYVGART, and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology to facilitate subcutaneous delivery of biologics. The product is to be administered subcutaneously by a healthcare professional as a single injection (1,008 mg fixed dose) over 30-90 seconds in cycles of once weekly injections for four weeks.
“The MG Community is energized and excited about another effective FDA-approved treatment available for those diagnosed with generalized MG. Thank you to our industry partner, argenx,” said Samantha Masterson, president and chief executive officer at the Myasthenia Gravis Foundation of America. “The ease and convenience of a subcutaneous injectable treatment will undoubtedly be very well-received by the patient community because the treatment process fits into the daily lives of patients around the country. Patients have an individualized approach and another important option for managing their personally unique symptoms of MG.”
VYVGART Hytrulo is expected to be available for patients in the United States in July 2023. argenx is committed to supporting access for patients to its medicines and has decided to price VYVGART Hytrulo at parity to VYVGART on a net annual revenue basis.
For more information, you can read the news release. Check out the official website brought to you by argenx, vyvgarthytrulo.com.
Myasthenia Gravis Foundation of America (MGFA) Announces Official Start of MG Awareness Month Around the World
MGFA News Release – Myasthenia.org
June 1, 2023
Global myasthenia gravis community taking action in June to spread awareness about rare disease and highlight
debilitating challenges experienced by patients. The MG Community around the world is turning Awareness into Action in June to educate everyone about the challenges of the rare neuromuscular disease myasthenia gravis (MG).
Dr. James Howard on Complications with Myasthenia Gravis
AJMC.com
May 26, 2023
Current myasthenia gravis therapies can create other complications, said James F. Howard Jr, MD, professor of neurology at the University of North Carolina at Chapel Hill, former chief of the Neuromuscular Disorders Division, and former James F. Howard Distinguished Professor of Neuromuscular Disease.
Nowak Receives Impact Award from the MGFA
Yale School of Medicine Website
April 12, 2023
The Myasthenia Gravis Foundation of America (MGFA) has awarded its Impact Award to Richard Nowak, MD for his exceptional leadership, collaboration, innovation, and dedication to advancing the mission of the MGFA. Dr. Nowak was formally recognized at the 2023 MGFA National Patient Conference in New Orleans, La.
Patients Rising Director Stresses Importance of Self-advocacy
Myasthenia Gravis News
April 4, 2023
“The first thing I really need to let you know is that you’re the only person who can really make yourself empowered,” Jim Sliney Jr. said in his presentation at the MG Foundation of America (MGFA) National Patient Conference, held March 26-28 in New Orleans.
‘Life-hacking,’ self-care, and staying active at core of living with MG
Myasthenia Gravis News
April 4, 2023
In a workshop at the MG Foundation of America (MGFA) National Patient Conference, a group of panelists discussed how MG has impacted them, and shared some tips and strategies that help them navigate life with the disease. The conference took place March 26–28 in New Orleans.
Hope called a ‘remedy for fear’ and powerful tool for managing MG
Myasthenia Gravis News
March 30, 2023
Hope can help improve a person’ quality of life and may even modulate disease activity, according to Jeffrey Rosenfeld, MD, PhD, director of the Center for Restorative Neurology at Loma Linda University Health, who discussed the topic at the MG Foundation of America (MGFA) National Patient Conference, March 26-28, in New Orleans. Rosenfeld’s presentation was titled, “The Power of Hope for the MG patient.”
Clinicoserological Insights into Patients with Immune Checkpoint Inhibitor-induced Myasthenia Gravis
Annals of Clinical and Translational Neurology
March 2023
Gianvito Masi, MD and Kevin C O’Connor, PhD
Cases of myasthenia gravis (MG) have been recently described as rare but life-threatening adverse events following the administration of immune checkpoint inhibitors (ICI), a novel type of cancer immunotherapy. Patients with ICI-MG often test positive for acetylcholine receptor (AChR) autoantibodies, but unlike idiopathic MG, the role of AChR autoantibodies in ICI-MG pathology is unknown. To address this, we studied a cohort of ICI-MG patients by functionally profiling their AChR autoantibodies. We found that a subset of patients may harbor AChR autoantibodies with molecular features similar to those of idiopathic MG. In other cases, however, such autoantibodies lack overt pathogenic potential, suggesting alternative factors as key mediators of disease. These findings have direct clinical implications, as they challenge the role of AChR autoantibody testing in establishing definite ICI-MG diagnoses and corroborate the need for a thorough assessment when evaluating ICI-related adverse events.
Read entire published paper HERE.
Understanding Myasthenia Gravis: SoCal resources available for those affected
Desert Health News
March 2023
Imagine having people think you’re too serious because you don’t smile often. What they don’t know is that weakness of your facial muscles prevents you from easily curving your mouth upward. This is just one of the symptoms you might experience if you have myasthenia gravis (MG). This rare, neuromuscular, autoimmune disease has been diagnosed in more than 70,000 people in the United States – and likely more remain undiagnosed.
Myasthenia Gravis Foundation of America (MGFA) Announces Alliance with Patients Rising to Offer MG Patient Advocacy and Support Services
MGFA News Release – MGFA Website
February 27, 2023
Myasthenia Gravis Foundation of America is aligning with Patients Rising, a Washington D.C.-based non-profit that provides education, resources, and advocacy for people living with chronic and life-threatening illnesses. Patients living with myasthenia gravis (MG) will benefit from The Patient Helpline for support services as well as educational patient advocacy and legislative training.
Redding sisters fighting rare disease
WKRC 7 ABC
February 20, 2023
Stephanie Madole’s two daughters, ages six and nine, wake up with a life-threatening disease daily. Raised in Redding, nine-year-old Elizabeth and six-year-old Charlotte both have Myasthenia Gravis.
Doctors at MCG/AU Health lead the charge in clinical trials for rare autoimmune disease
WJBF 6 ABC
February 10, 2023
Doctors at AU Health are leading the charge in clinical trials to find treatment options for a rare autoimmune disease. When our immune systems detect something harmful, they create antibodies.
Using the Myasthenia Gravis Patient Registry Management for Research Studies: Paul Strumph, MD
Neurology Live
February 9, 2023
Paul Strumph, MD
Recently, the Myasthenia Gravis Foundation of America (MGFA) re-launched its MGFA Global MG Patient Registry to combat these challenges for patients. In a recent interview, Paul Strumph, MD, chief medical officer at Seraxis Pharmaceuticals, and patient with MG, sat down with NeurologyLive® to discuss the attributes of the MGFA’s patient registry from a research focused perspective. He spoke on how patients can retrieve information about research studies through the registry and it how it helps researchers identify the right patients. Strumph, lead of the MGFA Global MG Patient Registry, also talked about the registry is managed by the organization with the goal of generating research.
Significance of Relaunched Patient Registry for Myasthenia Gravis Research: Richard Nowak, MD, MS
Neurology Live
February 7, 2023
Richard Nowak, MD, MS
The MGFA partnered with Alira Health to re-launch its MGFA Global MG Patient Registry. The registry allows patients with MG to submit their health data in a secure portal, thus facilitating research to gain more knowledge on the disease, improve patient outcomes, and possibly explore more effective treatments for MG. Richard Nowak, MD, MS, assistant professor of neurology, Yale School of Medicine, sat down with NeurologyLive® in an interview to talk about the relaunch of MGFA’s patient registry. Nowak, who also serves as MGFA’s chief medical advisor, spoke about the implications and the significance of research with using the registry to recruit patients in clinical trials.
Rare Disease Awareness Month – Houston
KHOU 11 Houston
February 1, 2023
Dr. Aziz Shaibani joins Great Day Houston to give insight on two rare diseases, Stiff Person Syndrome and Myasthenia Gravis.
Myasthenia Gravis Foundation of America (MGFA) Re-launches the MGFA Global MG Patient Registry with Partner Alira HealthSource
MGFA News Release – MGFA Website
January 25, 2023
Safe, secure registry utilizes MG patient data submissions to drive new research and discoveries for better treatments and potentially a cure for myasthenia gravis. The MGFA Global MG Patient Registry enables MG patients to submit their health and symptom data to be securely used to design and drive new research studies and clinical trials to find better treatments and potentially a cure for MG.
Penn Medicine Researchers Take Step Toward Development of Personalized, CAR T-like Treatment for Rare Form of Autoimmune Disease Myasthenia Gravis
Penn Medicine News
January 20, 2023
The revolutionary chimeric antigen receptor (CAR) T cell therapy, used to treat patients with certain types of cancers, can be reworked to treat a form of the autoimmune disease myasthenia gravis (MG), according to a preclinical study conducted in a small animal model from the Perelman School of Medicine at the University of Pennsylvania and published in Nature Biotechnology.
2022 COVERAGE
FDA Accepts NDA for Myasthenia Gravis Treatment Zilucoplan
Neurology Live
November 14, 2022
According to an announcement, the FDA has accepted the new drug application (NDA) of UCB Pharma’s investigational, subcutaneously delivered agent zilucoplan for the treatment of adults with acetylcholine receptor antibody positive (AChR-Ab+) generalized myasthenia gravis (gMG). The news comes shortly after the European Medicines Agency validated the Marketing Authorization Application (MAA) of zilucoplan for the same indication.
What You Need to Know about the Autoimmune Disease Myasthenia Gravis
The Monitor – Vida Health
October 13, 2022
You might have read about Ramsey Muniz in Monday’s edition of The Monitor. You might have seen the television advertisement for a medication specifically developed for treating Myasthenia Gravis. Beyond this, mention of the disease is minimal. Many have not heard of MG, or known the symptoms.
‘This Project Kept Me Alive’: Jazz Guitarist John Stein Returns to Stage after Illness
The Patriot Ledger
November 3, 2022
John Stein’s new double-album “Lifeline” began as a necessary adjustment to a serious health issue, but it has become an acclaimed testament to the guitarist’s multidecade music career.
Myasthenia Gravis Patient Wants Change in her Lifetime
KOLO Reno Nevada
June 20, 2022
At age 12 Rachel Blinn was diagnosed with Myasthenia Gravis. Then and now it is a rare disease with children.
She said she and her family knew something was wrong the day Rachel performed in a school play.
NORD Applauds House Passage of HR 7667, the Food and Drug Amendments of 2022
The US House of Representatives passed by a vote of 392-28 the Food and Drug Amendments of 2022 (HR 7667), legislation with significant implications for millions of Americans living with rare diseases. The bipartisan bill is an important step forward in reauthorizing the FDA’s medical product user fee programs, helps strengthen the accelerated approval pathway, and protects the intent of the Orphan Drug Act.
“On behalf of millions of Americans living with rare diseases, we commend House lawmakers for working in a bipartisan way to pass the critically important Food and Drug Amendments of 2022,” said Heidi Ross, Vice President, Policy & Regulatory Affairs, NORD. “It is clear from this vote that our elected officials have heard our community’s calls for action, and they recognize the importance of these user fee acts in helping to address the urgent needs of their constituents who are affected by rare diseases.”
READ THE FULL PRESS RELEASE ONLINE
Colorado Becomes 24th State to Establish a Rare Disease Advisory Council (RDAC)
Governor Jared Polis of Colorado, signed Senate Bill 186 (SB 186) into law to create a Rare Disease Advisory Council, making Colorado the 10th state to establish an RDAC since the start of 2021.
“Thank you to the bill sponsors, stakeholders, and everyone in the rare disease community for helping to establish a Rare Disease Advisory Council here in Colorado. This RDAC will have a positive impact in making meaningful improvements to the everyday lives of families like mine. I’m excited about the future and optimistic we can fight rare disease together with this council,” said Nick Kirchhof, NORD Colorado Rare Action Network Volunteer State Ambassador.
READ THE NEWS ABOUT THIS NEW RARE DISEASE ADVISORY COUNCIL
AstraZenenca/Alexion’s Ultomiris Treatment Approved in the US for Adults with Generalized Myasthenia Gravis
AstraZeneca Website
April 28, 2022
Our industry partner AstraZeneca, and its Alexion rare disease group, announced that the United States Food & Drug Administration (FDA) has officially approved the Ultomiris (ravulizumab-cwvz) treatment for adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive, which represents 80% of people living with the disease. Ultomiris showed early effect and lasting improvement in activities of daily living and has potential to reduce treatment burden with dosing every 8 weeks
Samantha Masterson, President & Chief Executive Officer, Myasthenia Gravis Foundation of America (MGFA), said: “gMG takes a physical and emotional toll on those living with the disease. We are grateful for continued innovation and research into new treatment and dosing options to meet the needs of more patients and reduce the treatment burden. With the approval of Ultomiris, we’re excited that MG patients now have another option to consider as part of their personalised treatment strategies that may offer more convenience and improve muscle weakness.”
Read more about this critical new treatment.
Safety and tolerability of SARS-Cov2 vaccination in patients with myasthenia gravis – Vaccination Strongly Recommended
NIH Publications
April 7, 2022
During the COVID-19 pandemic, Myasthenia gravis (MG) patients have been identified as subjects at high-risk of developing severe COVID-19, and thus were offered vaccination with priority. The lack of direct data on the safety and tolerability of SARS-CoV-2 vaccines in MG have contributed to vaccine hesitancy. To address this issue, the safety and tolerability of SARS-CoV-2 vaccines was assessed in a large cohort of MG patients from two referral centers.
Patients with confirmed MG diagnosis, consecutively seen between October and December 2021 in two MG centers were enrolled. Demographics, clinical characteristics, and information regarding SARS-CoV-2 infection/vaccination were extracted from medical reports and/or collected throughout telephonic or in person interviews.
Our data support the safety and tolerability of mRNA-COVID-19 vaccines, which should be strongly recommended in MG patients who could be at higher risk of complications if exposed to SARS-CoV-2 infection.
Review the published research in Doc Wire News.
Read the full report in European Journal of Neurology.
Latest MGFA-Supported MG Research – Adverse Event Unit Project
PLOS ONE
March 29, 2022
Dr. Michael Hehir has shared and published the first paper from the Adverse Event Unit project. This important MG research was published in PLOS ONE. The MGFA supported and provided early funding for the project process. This paper represents the first step in important efforts to understand the adverse event burden of the treatments medical professionals use for patients with myasthenia gravis and other neurological disorders.This project was initially supported by the 2016 MGFA/AAN/ABF Clinician Scientist Development Award.
Read the RESEARCH PROJECT DOCUMENT.
Read the research.
First successful bone marrow transplant for multi autoimmune syndrome in Iran
Tehran Times
February 15, 2022
Specifically, her myasthenia gravis was a refractory one with severe muscle weakness, resulting in repeated respiratory failures that required her to be intubated and mechanical ventilation at the ICU. However, the transplant team decided to perform such a treatment for the first time in Iran. Spending three months inspecting this demanding multidimensional job, ultimately autologous bone marrow transplant was chosen as the most effective approach to take.
Myasthenia Gravis Foundation of America (MGFA) Announces Annual Volunteer Awards at 2022 National Patient Conference
February 11, 2022
MGFA National Patient Conference is the largest online gathering of the myasthenia disease community: Prestigious volunteer awards recognize MG Community members for their impact and commitment.
The Myasthenia Gravis Foundation of America (MGFA), the largest, leading patient advocacy organization solely dedicated to finding a cure for the rare neuromuscular disease myasthenia gravis (MG) while improving the lives of those living with MG, announced today the recipients of its annual MGFA Volunteer Awards at the 2022 National Patient Conference.These unique volunteers and organizations have been recognized for their deep commitment to the MGFA and the MG community through patient advocacy, unprecedented medical advancements, and positive impact for those living with MG and caregivers as well as everyone across the community.
2021 COVERAGE
argenx Announces U.S. Food and Drug Administration (FDA) Approval of VYVGART (efgartigimod alfa-fcab) in Generalized Myasthenia Gravis
December 17, 2021
argenx SE (Euronext & Nasdaq: ARGX) announced that the U.S. Food and Drug Administration (FDA) has approved VYVGART (efgartigimod alfa-fcab) for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. These patients represent approximately 85% of the total gMG population. With this regulatory milestone, VYVGART is the first-and-only FDA-approved neonatal Fc receptor (FcRn) blocker.
“The gMG community has long-awaited the FDA approval of VYVGART, especially for those patients who struggle with basic personal tasks such as speaking, chewing and swallowing food, brushing teeth and hair, and in some severe cases, breathing,” commented Samantha Masterson, President and Chief Executive Officer of the Myasthenia Gravis Foundation of America. “We thank argenx for its continued commitment to the gMG patient community, which led them to deliver this much-needed new treatment option with the potential to change the lives of many gMG patients.”
UCB announces positive Phase 3 results for rozanolixizumab in generalized myasthenia gravis
UCB, a global biopharmaceutical company, today announced positive topline results from the Phase 3 MycarinG study1 evaluating rozanolixizumab, a subcutaneously (SC) infused monoclonal antibody targeting the neonatal Fc receptor (FcRn), versus placebo in adults with generalized myasthenia gravis (gMG). Overall rozanolixizumab was well tolerated and no new safety signals were identified. “For the many thousands of people living with myasthenia gravis around the world, current treatment options can be very limited,” said Samantha Masterson, Chief Executive Officer of the Myasthenia Gravis Foundation of America (MGFA). “Given that this disease causes a wide range of symptoms, some of which can require urgent intervention or hospitalization, there is a critical need for new treatment options that could address the unmet needs of patients living with myasthenia gravis.”
MGFA Poised for 2021 Coast-to-Coast Walk 2740 Challenge
Myasthenia Gravis News
September 16, 2021
With the goal of raising funds and awareness to improve care and find a cure for myasthenia gravis (MG), the Myasthenia Gravis Foundation of America (MGFA) is hosting the 2021 Coast-to-Coast Walk and 2740 Challenge on Nov. 13.
Australian wheelchair rugby player overcomes rare disease to be in the Paralympics
Tokyo Paralympic.org
August 27, 2021
Richard Voris was left out of Australia’s wheelchair rugby team that won the Rio 2016 gold medal, despite being a regular in the squad since 2013. The reason was an autoimmune disease called Myasthenia Gravis. But he is back!
Unstoppable Glenda Thomas Made Incredible Accomplishments Despite her MG
Authority Magazine
July 12, 2021
As part of our “Unstoppable” series, I had the pleasure of interviewing Glenda Thomas. She was diagnosed with MuSK myasthenia gravis (MG) and has been an active volunteer for the Myasthenia Gravis Foundation of America
Together We are Turning Awareness into Action
Metropolis Planet
June 16, 2021
In fact, at that time, I had never even heard of Myasthenia Gravis (MG). Now, during the month of June, we are #MGSTRONG, and together, we “Turn MG Awareness into Action.”
Framingham’s Glenda Thomas Named to Serve on State Rare Disease Board
MetroWest Daily News
June 7, 2021
The Framingham resident recently added another title to her ever-growing resume. Thomas was recently appointed to the newly formed Rare Disease Advisory Council of Massachusetts.
MGFA Events Set to ‘Turn Awareness Into Action’ Throughout June
Myasthenia Gravis News
June 1, 2021
MG Action Month — Turning Awareness into Action is this year’s theme. MGFA has a host of events and activities planned, including a live, virtual interactive map that showcases individual stories, with states and countries “lighting up” to indicate Awareness Month activity. The organization is inviting the entire MG community to participate.
NEWS RELEASE: Glenda Thomas Selected as Member of the Rare Disease Advisory Council of Massachusetts
May 25, 2021
Glenda Thomas, an MG advocate, patient, and Bay State resident who has been an extremely active volunteer, has been appointed as the first-ever MG advocate to the newly-established Rare Disease Advisory Council of Massachusetts. Glenda will be publicly sworn in during an online ceremony on Thursday, May 27, 2021 at 11:30 a.m Eastern. Read the News Release to access the zoom link for the online ceremony.
Comparison Between Rituximab Treatment for New-Onset Generalized Myasthenia Gravis
JAMA Neurology
May 4, 2021
Does the response to rituximab differ between patients with new-onset vs refractory generalized myasthenia gravis, and how does rituximab compare with conventional immunotherapy in these patients?
April 11, 2021
Largest Patient Advocacy Organization Solely Committed to Myasthenia Selects MG Community Members for Prestigious Awards that Recognize Impact, Advocacy, and Dedication
Her Eyelid Drooped and She Kept Getting Weaker. What Was Going On?
New York Times Magazine
March 4, 2021
Dozens of tests turned up nothing. Then a specialist had a theory. It was myasthenia gravis.
MG Patients Should Be Priority for COVID-19 Vaccine, Report Says, Citing Risks
Myasthenia Gravis News
March 2, 2021
University of Arkansas data suggests MG patients should be positioned higher in the eligibility list to receive the COVID-19 vaccines.
Secrets to Living Longer and Feeling Healthy with Plant-based Diet
The Beet
February 13. 2021
MG patient Fran German and husband switch to plant-based diet and feel younger and healthier.
National Organization for Rare Disorders (NORD) Adds MG to RareCare Support Network
NORD Website
January 24, 2021
NORD has added myasthenia to its RareCare support network. The network provides assistance programs to help patients obtain life-saving or life-sustaining medication they could not otherwise afford. https://rarediseases.org/for-patients-and-families/help-access-medications/patient-assistance-programs-2/ You can find out more about the MG Patient Assistance Programs and other supportive patient assistance from NORD HERE: bit.ly/NORDAssistance.
argenx Officially Files for FDA approval of Efgartigimod for Generalized MG
Myasthenia Gravis News
January 12, 2021
argenx has officially files for U.S. FDA approval of Efgartigimod for generalized MG.
Double-seropositive myasthenia gravis: a distinct subtype?
Neurological Sciences
January 12, 2021
This study investigated the characteristics of double-seropositive myasthenia gravis (DSP-MG) in southern China for disease subtype classification.
2020 Coverage
AstraZeneca Agrees to Acquire Alexion, the Maker of Soliris, for $39 Billion
Alexion.com
December 12, 2020
Alexion, a Boston-based rare disease pharmaceutical and MGFA industry partner, has entered into an agreement to be acquired by AstraZeneca, one of the world’s largest drug companies. READ MORE
My Cause, My Cleats: Dexter Williams of the Green Bay Packers Supports MG Awarenesss
Packers.com/Sports Illustrated
December 6, 2020
Dexter Williams of the NFL Green Bay Packers decorates his cleats to direct attention to those with myasthenia during annual “My Cause, My Cleats” initiative. READ MORE
International Experts Update, Expand MG Management Guidelines
Myasthenia Gravis News
December 4, 2020
A panel of 16 international experts, initially selected by MGFA, has updated and expanded recommendations for best management of myasthenia gravis (MG). READ MORE
MDA Wants Neuromuscular Disease Patients to have Early Access to COVID-19 Vaccine
Myasthenia Gravis News
December 4, 2020
The Muscular Dystrophy Association is encouraging the U.S. CDC to recommend that people living with neuromuscular diseases (NMDs) such as myasthenia have early access to any federally approved COVID-19 vaccine. READ MORE
Plasma Shortage Intensified Because of COVID-19
The Morgan Hill Times
October 7, 2020
With the Covid-19 pandemic cancelling many donation drives throughout the nation, the Myasthenia Gravis Foundation of America is reporting a severe shortage of plasma donations. Read interview with volunteer Deborah Vick. READ MORE
Research survey for rare disease patients and their families about impacts of COVID-19
MGFA Published Survey
April, 2020
How is the novel coronavirus pandemic impacting people with rare diseases and their families? Complete the 20-minute research survey from home or learn more at this link. The Rare Diseases Clinical Research Network (RDCRN) is conducting this study. The network is funded by the National Institutes of Health. It includes 23 research teams working to advance diagnosis and treatment of groups of rare diseases. The Myasthenia Gravis Foundation of America and Conquer MG partners with RDCRN through its work with the Myasthenia Gravis Rare Disease Network (MGNet).
Guidance for the management of Myasthenia Gravis (MG) and Lambert-Eaton Myasthenic Syndrome (LEMS) during the COVID-19 pandemic
International MG/COVID Working Group
March 23, 2020
International MG/COVID-19 Working Group1SaijuJacoba2SrikanthMuppidib2AmandaGuidoncJeffreyGuptilldMichaelHehireJames F.HowardJrfIsabelIllagRenatoMantegazzahHiroyukiMuraiiKimiakiUtsugisawajJohnVissingkHeinzWiendllRichard J.Nowakm2
Coronavirus Disease 2019 (COVID-19) is a new illness caused by a novel coronavirus, severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). Symptoms are variable but typically include fever, cough, respiratory symptoms, diarrhea, reduction of smell and taste sensation. Severity ranges from mild to severe and the virus may lead to pneumonia, acute respiratory distress syndrome and death, in some patients. Nearly every country in the world has been affected by this virus and is currently defined as a pandemic, by the World Health Organization. There are no known proven therapies for treating this virus and no vaccine to prevent the infection at this time.
The full guidance is available here.
MGNet Webinar- “Myasthenia Gravis and the COVID-19 Epidemic: Things for you to know”
MGFA Program
March 18, 2020
Presenters: Hana Axelrod, MD, MPH; Henry J. Kaminski, MD; Helen Girma, George Washington University Department of Neurology
We are pleased to share that the MGNet webinar, “MG and COVID-19: Things for you to Know” has been recorded and is available for viewing. We apologize for any technical difficulties in registering or participating in the webinar on March 18.
To view the recording of this webinar, please see here
MG Walks and COVID-19 (Coronavirus)
MGFA Fundraising News
March 2020
The health and safety of our participants, sponsors, volunteers and staff is our top concern. Currently, ALL spring walks from March 14-May 30 have been postponed until further notice. Additional details can also be found on our MG Walk website here.
We will look at potential dates for this fall and keep you updated as we learn more. If we are able to secure a fall date, then all donations will roll over to the new walk date.
**Any decisions made going forward will absolutely be dependent on where we are at with this global health crisis.
COVID-19 (Coronavirus)- General Information and Management
MGFA New Resources
March 2020
Due to the recent news regarding COVID-19 (coronavirus), we are providing some guidance around COVID-19, as well as reliable sources for information. See more information here.
MGFA announces 2020 research funding opportunities
We are pleased to announce the release of our Request for Applications for our High Impact Clinical Research and Scientific Pilot Projects on Myasthenia Gravis and Related Neuromuscular Junction Disorders.
We are also very pleased to announce our first targeted research opportunity to benefit the Seronegative MG community.
To learn about our research agenda and complete funding opportunities, see here.
University of California, San Francisco, Launches First Consortium on Pediatric MG in Partnership with MGFA and MDA
For families with children that have myasthenia gravis, we have some exciting news to share. Thanks to a generous gift from an anonymous donor to the University of California, San Francisco, the first clinical Pediatric Myasthenia Gravis Consortium is now a reality. MGFA was honored to provide a grant to underwrite the costs for the September inaugural meeting of this exciting new project, and to participate on the advisory committee for the Consortium.
GW Researchers Receive $7.8 Million to Establish Rare Disease Network for Myasthenia Gravis, MGNet, supported by commitments from MGFA and Conquer MG
MGFA is proud to represent the MG Community as a member of MG Net, and has committed $250,000 of funding ($50,000 for each year) to support the project. This commitment from MGFA, as well as that of Illinois-based Conquer MG, was instrumental in demonstrating the support of the MG Community for the project—an essential component of the criteria for funding established by the NIH. Press release available here.
The National Institutes of Health (NIH) has awarded a research team at the George Washington University (GW) $7.8 million to establish a rare disease network for myasthenia gravis. The network, which will be part of 25 established NIH Rare Diseases Clinical Research Networks, will include basic and clinical investigators, patient advocacy groups and biotechnology and pharmaceutical companies working together to enhance therapeutic development for this rare disease. The team is led by former and current MGFA Medical and Scientific Advisory (MSAB) Chairs, Henry Kaminski, MD and Linda Kusner, PhD, and the steering committee members are all leaders of the MGFA MSAB as well.
The grant will fund research into the underlying pathophysiology of the disease, provide fellowships in MG for young investigators, and fund pilot grants. This funding will also ensure that the serum bank created by the MGFA’s transformative grant will continue.
- Clinical Research Funding Announcement: PIlot Grant Program for Myasthenia Gravis
- Myasthenia Gravis Network (MGNet) Scholar Program Research Funding Announcement
MGFA’s 2020 Virtual Conference, April 5-7- Recorded Presentations Available Here
To view recorded presentations, resources and sponsor information, please complete a brief registration form and visit our virtual conference space here. Please see some helpful tips for navigating our virtual conference space here.