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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 AOC 1044 Data
Avidity Biosciences, which received early funding from CureDuchenne, announced positive initial data from their EXPLORE44 […]
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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 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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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 […]
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I’m at a crossroads in life, once again
Starting last week, I’ve been trying to get my life back on track after enduring […]
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Phase 1/2 update ‘encouraging’ for DMD gene therapy RGX-202
The experimental gene therapy RGX-202 has been found to be well tolerated at a high […]
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Your Citizen Science At Work
By sharing your anonymous data through The Duchenne Registry, you are strengthening the power of […]
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PGN-ED051, skipping exon 51, showing benefits in Phase 2 trial
A low dose of PGN-ED051, PepGen’s investigational exon 51-skipping therapy, safely increased dystrophin protein levels […]
