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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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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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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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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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Entrada Therapeutics announces the FDA has removed the clinical hold on ENTR-601-44, their experimental therapeutic designed to skip exon 44
As an early funder of Entrada Therapeutics, we are pleased to share that the FDA […]
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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, […]
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Entrada Therapeutics Announces FDA Removal of Clinical Hold on ENTR-601-44
Entrada Therapeutics, Inc. has announced that the U.S. Food and Drug Administration (FDA) has lifted […]
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Solid Biosciences Reports Positive Initial Clinical Data
Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003 Solid […]
