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Oral therapy moves to follow-up after strong data in DMD adults
Dosing has begun in a long-term follow-up study testing Satellos Bioscience’s experimental oral therapy SAT-3247
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PPMD Provides $400,000 in Funding to MyoGene Bio Through PPMD Venture Pathways Program to Support Development of Gene Editing Platform
PPMD and MyoGene Bio (MyoGene) are excited to announce that PPMD has provided $400,000 in
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Sarepta Announces Completion of ESSENCE Trial for Exon 45 & 53 Skipping Therapies
Sarepta Therapeutics has announced top-line results from the ESSENCE trial, a global, randomized double-blind, placebo-controlled
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Sarepta’s Amondys 45 and Vyondys 53 confirmatory trial fails to achieve statistical significance on primary endpoint
Sarepta has completed the ESSENCE study, which is a confirmatory trial aimed at supporting full
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Solid plans to seek FDA guidance in 2026 for Duchenne Gene Therapy Candidate SGT-003 S
Solid Biosciences today reported an update on the progress of the INSPIRE DUCHENNE open label,
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Books to Add to Your Holiday Shopping List This Year: Spotlight on Community Authors
In August, Quest had the privilege of spotlighting published authors living with neuromuscular disease and
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I’m learning to accept that grief is part of my DMD caregiver journey
Last weekend, my husband and I cleaned out our storage unit. My husband loves that
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Novartis pays $12B for Avidity and its muscle-targeting RNA platform
In a deal worth roughly $12 billion, pharmaceutical giant Novartis has agreed to acquire Avidity
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RegenXBio completed enrollment in pivotal gene therapy trial; still enrolling the confirmatory trial ages 1 year and above
RegenXBio announced that they have completed enrollment in the Phase 1/2 portion of their RGX-202
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Getting Ready for the 2026 MDA Clinical & Scientific Conference
It’s that time of year when we come together to connect, share, learn—while accelerating progress
