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Adjusting my definition of what it means to be a man
Kevin Schaefer, the associate director of community content at Bionews, the parent company of this […]
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SAT-3247 for DMD wins FDA rare pediatric disease designation
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to SAT-3247, […]
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Finding Support to Manage Behaviors in Duchenne and Becker
Research has shown that dystrophin is usually present in the brain as well as in […]
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Solid Biosciences has begun dosing patients in their clinical study of their next-generation gene therapy, SGT-003 for those living with Duchenne.
Letter to the Duchenne Community – INSPIRE DUCHENNE update Dear Duchenne Community, We are writing […]
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PPMD Urges FDA to Conduct a Full Review of Ataluren (Translarna) Following Resubmission of NDA
PTC Therapeutics has shared that it has resubmitted its New Drug Application (NDA) to the […]
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Avidity Biosciences Announces Positive Data From Phase 1/2 EXPLORE44™ Trial of AOC 1044 in Individuals Amenable to Exon 44 Skipping
Avidity Biosciences, Inc. today announced positive data from the company’s Phase 1/2 EXPLORE44 clinical trial […]
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The dance floor at my daughter’s wedding had room for all
I’m officially a mother-in-law! That’s a new title for me. I’m a mom to the […]
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Avidity Biosciences Announces Positive AOC 1044 Data
Avidity Biosciences, which received early funding from CureDuchenne, announced positive initial data from their EXPLORE44 […]
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WATCH: Edgewise Therapeutics – Clinical Trials in Becker Muscular Dystrophy (Webinar)
Edgewise Therapeutics recently joined PPMD for a webinar to provide the Becker community information about […]
