Tag: Rare Disease

News, Research

2022 Clinical & Scientific Conference March 15 Highlights

Sujatha Gurunathan

Today was the second full day of sessions of 2022 MDA’s Clinical & Scientific Conference. The three major themes of today’s sessions were (1) therapeutic strategies to treat NMDs, (2) clinical trial design and implementation, and (3) optimizing patient care.  Highlights Mitochondrial interventions: small molecule and gene therapy Carlos Moraes, PhD, from the University of . . .

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Kids, Young Adults, Adults, Research, Living with Muscular Dystrophy

Simply Stated: What is Duchenne Muscular Dystrophy?

Sujatha Gurunathan

Duchenne muscular dystrophy (DMD) belongs to a group of rare genetic disorders, known as dystrophinopathies, that are caused by mutations of the dystrophin gene. DMD is characterized by progressive degeneration and weakness of the body’s voluntary muscles, primarily the skeletal muscles that control movement. In later stages, the heart and respiratory muscles may also be . . .

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