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Press Releases and MGFA News
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
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.
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.
Myasthenia Gravis Foundation of America (MGFA) Announces Official Start of MG Awareness Month Around the World
MGFA News Release
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).
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.
Myasthenia Gravis Foundation of America Announces Alliance with Patients Rising to Offer MG Patient Advocacy and Support Services
MGFA News Release
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.
Using the Myasthenia Gravis Patient Registry Management for Research Studies: Paul Strumph, MD
Neurology Live
February 9, 2023
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
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.
MGFA Re-launches the MGFA Global MG Patient Registry with Partner Alira HealthSource
MGFA News Release
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.
GW Researchers Receive $7.8 Million to Establish Rare Disease Network for Myasthenia Gravis, MGNet, supported by commitments from MGFA and Conquer MG
George Washington University website
October 2, 2019
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
Myasthenia Gravis in the News
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.
NMD Pharma Successfully Completes First Phase of NMD670 in MG Clinical Study – Phase 2 Active and Recruiting
Science Translational Medicine
March 20, 2024
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.
NMD has opened phase 2 and is currently active and recruiting MG patients. Visit the study page on ClinicalTrials.gov to determine your eligibility and how to apply to be part of this exciting trial.
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.
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.
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
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.
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 MGFA, share more about the study and its impact on the patient and research community.
Read more about this published research
Modifying Patient T-cells Creates Novel Approach to Treating Myasthenia Gravis and other Diseases
Cartesian Website
July 1, 2023
Cartesian Therapeutics 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
UCB 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
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.
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.”
For more information, you can read the news release. Check out the official website brought to you by argenx, vyvgarthytrulo.com.
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.
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.