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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
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Simply Stated: Updates in Neuropathy Ataxia and Retinitis Pigmentosa (NARP) Syndrome
Neuropathy ataxia and retinitis pigmentosa (NARP) syndrome is a rare, maternally-inherited condition caused by mutations
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My family caregivers are feeling the pressure
I have a growing list of limitations, as a result of my facioscapulohumeral muscular dystrophy
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BBP-418 improves walking ability in LGMD2i patients in Phase 3 trial
Use of the oral therapy BBP-418 appeared to improve walking ability and lung function in
