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Milestone Moment: A Look Back at the First Gene Therapy Trial for DMD
In 2006, MDA funded the first-ever gene therapy trial for Duchenne muscular dystrophy (DMD). This
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Why You Should Practice Shared Healthcare Decision-Making
Facing a neuromuscular disease diagnosis can be overwhelming, but the more that individuals and their
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From the Ski Slopes to the Spirit of Inclusion
When I was learning to ski as a child in Washington state, like anyone new
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Early preventive heart treatment extends survival in DMD: Study
Preventive treatment with standard heart medications — when given before the onset of cardiac troubles
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Simply Stated: Updates in Pompe Disease and Other Glycogen Storage Diseases
Glycogen storage diseases (GSDs) are a group of rare inherited conditions that occur when the
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FSHD progression requires me to lighten my music rig
I’ve played music since I was 5 years old. My first professional playing job was
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MDA’s Development Grants Support Promising Researchers Entering the Neuromuscular Field
Since its founding 75 years ago, MDA has been the nation’s largest nonprofit supporter of
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Kicking off fresh creative ventures through social media
Despite the significant health challenges I face as a person living with Duchenne muscular dystrophy,
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ENTR-601-44 trial for DMD adults amenable to exon 44 skipping OK’d
The U.S. Food and Drug Administration (FDA) has given Entrada Therapeutics permission to launch a
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How an emergency medical plan can benefit those of us with MD
“ICE” is a widely used acronym for “in case of emergency.” In today’s fast-paced world,
