The latest info for the MG community is right here!
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
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.”
You can read more about this critical new treatment at the link below.
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 actual full report in Wiley publishing and European Journal of Neurology
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.
You can also read the RESEARCH PROJECT DOCUMENT HERE.
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.
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.
READ MGFA's FULL NEWS RELEASE
argenx SE (Euronext & Nasdaq: ARGX) announced on December 17, 2021 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.”
Read the official press release:
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.”
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.
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!
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
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.”
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.
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.
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.
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?
NEWS RELEASE: Myasthenia Gravis Foundation of America Presents Annual Volunteer Awards at 2021 National Conference
April 11, 2021
Largest Patient Advocacy Organization Solely Committed to Myasthenia Selects MG Community Members for Prestigious Awards that Recognize Impact, Advocacy, and Dedication
New York Times Magazine
March 4, 2021
Dozens of tests turned up nothing. Then a specialist had a theory. It was myasthenia gravis.
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.
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
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.
Myasthenia Gravis News
January 12, 2021
argenx has officially files for U.S. FDA approval of Efgartigimod for generalized MG.
January 12, 2021
This study investigated the characteristics of double-seropositive myasthenia gravis (DSP-MG) in southern China for disease subtype classification.
AstraZeneca Agrees to Acquire Alexion, the Maker of Soliris, for $39 Billion
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
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
MGFA Published Survey
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).
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"
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
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
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.
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.
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.
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.
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Magazines & Newsletters
- Focus on MG magazine: Our Focus on MG magazine is published twice a year. The magazine highlights MGFA programs and activities around the country. You can find useful information including MG community updates, practical advice on how to manage MG as a patient, caregiver, or friend and much more. Read the most recent issue.
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