Archives
Clinical Trial Alert: Phase 3 Study of Pozelimab and Cemdisiran in Adults with gMG
Researchers at Regeneron are seeking adults who have symptomatic, generalized myasthenia gravis (gMG), presenting with anti-AChR antibodies or anti-LRP4 antibodies, to participate in a phase 3 clinical trial (NIMBLE) to evaluate…
What You Need to Know About Substance Use Disorder
The CDC estimates that 40.3 million individuals in America experienced Substance Use Disorder (SUD) in the year 2020. SUD, also commonly referred to as substance abuse or addiction, is a…
Tags: Community, Healthcare, Mental Health, Resources
FDA Approves Biogen’s Qalsody for Treatment of SOD1-ALS
On April 25, the US Food and Drug Administration (FDA) granted accelerated marketing approval of Qalsody (tofersen) for the treatment of amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s…
Our Amazing Opportunity to Improve Air Travel: 5 Things You Need to Know
From broken bones to broken wheelchairs, we know traveling by air can be a nightmare for people living with a disability. However, MDA and its grassroots advocates have a unique…
Quest Podcast: There is No Such Thing as Normal
Megan DeJarnett is an author, speaker, disability DEI educator, and inclusion advocate. Megan lives with SMA and is the organizational founder of No Such Thing, a company with a mission…
Tags: Community, Parenting, Quest Podcast
MDA Makes Connections at the University of Minnesota’s Muscle Research Lab Day
MDA Family and Clinical Support Specialist, Jordann Hager There are exciting transitions happening at the Muscular Dystrophy Association! As we move to being in-person at MDA Care Centers again, there…
Tags: Community, MDA Care Centers, Research
At MDA Conference, Pompe Disease Researchers Stressed Importance of Newborn Screening
At the MDA Clinical & Scientific Conference, researchers discussed how therapy advances make newborn screening for Pompe disease even more important.
Tags: Drug Development, Featured Content, Gene Therapy, MDA Clinical and Scientific Conference, Research, Research Advances
MDA Ambassador Guest Blog: My Volunteer Journey
MDA Ambassador Amy Curran MDA Ambassador Amy Curran lives with GNE Myopathy. She resides in the suburbs of Philadelphia, Pennsylvania with her partner, Jon, and her dog, Lexi. She holds…
National Volunteer Month: Why I Volunteer for MDA
April is National Volunteer Month! As MDA celebrates a month honoring the incredible volunteers who make our mission possible, we are excited to spotlight four dedicated individuals and families from…
Simply Stated: Elevated Enzymes
Elevated enzymes are a frequently encountered problem in general medical practice, but their meaning often isn’t so simple to discern. When they’re found with a neuromuscular disease, the situation can…
Tags: Healthcare, Simply Stated