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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 FDA approval of the exon-skipping PMOs Amondys 45 and Vyondys 53. The study did reinforce the favorable safety profile of the therapies, but did not achieve statistical significance on the primary endpoint (4-step ascend velocity at 96 weeks). What comes…
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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, Phase I/II clinical trial with SGT-003 and their planned regulatory discussions in 2026. 23 Pediatric participants have been dosed (1E14vg/kg) so far in the INSPIRE DUCHENNE trial (data cutoff 31st October). SGT-003 was generally well tolerated, there were no cases of…
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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 sharing their books in celebration of National Read A Book Day. With so many talented and accomplished writers in our community and holiday shopping right around the corner, the second installment of our Spotlight on Community Authors blog series is…
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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 unit, while I feel like we need one so that we don’t accumulate too much stuff around the house. We started renting it two years ago when we were hosting our oldest son’s high school graduation party in the garage.…
