Archives
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
Inclusion and Accessibility for All: Understanding Your Rights as a Shopper
For those living with physical disabilities, accessibility is a cornerstone of inclusion. While a focus on inclusivity and diversity has become more prevalent in the last ten years, with many…
Tags: Community
Simply Stated: Research Updates in Pompe Disease
Pompe disease (also called acid maltase deficiency) is a rare, metabolic muscle disorder that causes slow, progressive muscle weakness, especially of the respiratory (breathing) muscles and those of the hips,…
MDA Peer Connections Program Creates Lasting Friendships
The MDA Peer Connections Program provides an opportunity for members of the neuromuscular disease community to build bonds with one another, both across the country and in their own neighborhoods.…
2023 MDA Clinical and Scientific Conference Starts Strong
The first day of the 2023 MDA Clinical & Scientific Conference covered FDA approval, gene therapy, ultra-rare diseases, and more.
Tags: Clinical Trials, Drug Development, Featured Content, Gene Therapy, Innovation, MDA Clinical and Scientific Conference, Research
MDA Ambassador Guest Blog: The Magic of MDA Summer Camp
MDA Ambassador Leticia Tatum MDA Ambassador Leticia Tatum, 42, of Birmingham, Alabama was diagnosed with SMA at the age of two. She currently serves as the Vice President of Human…
Quest Podcast: The Changing Landscape of Neuromuscular Care
As new treatments, cutting edge research, and a better understanding of genetic sequencing and mutations bring innovative change to the treatment landscape for neuromuscular disease, understanding your options and accessing…
Tags: Drug Development, Gene Therapy, Genetic Testing, Healthcare, Innovation, MDA Care Centers, My Gene Therapy Journey, Quest Podcast, Research, Research Advances