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Blog Post | Science + 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


Blog Post | Get Involved

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…

Tags: Ambassador Guest Blog, Ambassadors, Community, Volunteers, Year of the Volunteer


Blog Post | Get Involved

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…

Tags: Community, Fill the Boot, Fundraising, Summer Camp, Volunteers, Year of the Volunteer


Blog Post | Finding a Diagnosis, Health, Science + Research

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


Blog Post | Advocacy, Inclusion

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


Blog Post | Finding a Diagnosis, Science + Research

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,…

Tags: Clinical Trials, Drug Development, Gene Therapy, Simply Stated


Blog Post | Finding a Diagnosis, Get Involved, Lifestyle

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.…

Tags: Community, MDA Peer Connections, Relationships


Blog Post | Science + Research

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


Blog Post | Lifestyle, Personal Stories

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…

Tags: Ambassador Guest Blog, Ambassadors, Summer Camp


Blog Post | Health, Science + Research

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