September 13, 2022

Sarepta Shares EMBARK Study (SRP-9001-301) Enrollment Update

Sarepta Therapeutics, in partnership with Roche, has announced the EMBARK study of delandistrogene moxeparvovec (SRP-9001) in participants with Duchenne is now fully enrolled. Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy intended to deliver…

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September 12, 2022

Edgewise Announces 4-Month Interim Results from ARCH Open Label Study of EDG-5506 in Adults with Becker

Edgewise Therapeutics has released 4-month interim results from the ongoing ARCH open label study of EDG-5506 in adults with Becker muscular dystrophy. EDG-5506 is a small molecule, taken orally, that is intended to help protect muscle…

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September 7, 2022

Edgewise Announces FDA Authorization for Phase 2 Clinical Trial of EDG-5506 in Participants with Duchenne

Edgewise Therapeutics has announced the FDA has authorized the initiation of the LYNX Phase 2 clinical trial of EDG-5506 in participants with Duchenne. EDG-5506 is a small molecule, taken orally, that is intended to help protect muscle by reducing…

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September 6, 2022

Dyne Therapeutics Announces First Patient Dosed in Phase 1/2 DELIVER Clinical Trial of DYNE-251

Dyne has announced that the first participant has been dosed in the Phase 1/2 DELIVER clinical trial of DYNE-251 in participants with Duchenne amenable to skipping exon 51. Dyne-251 is an exon skipping product that combines…

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September 6, 2022

Sarepta Therapeutics Announces That FDA has Lifted its Clinical Hold on SRP-5051

Sarepta Therapeutics has announced that the FDA has lifted the clinical hold on SRP-5051 (vesleteplirsen) following a serious adverse event of hypomagnesemia (low magnesium). SRP-5051 is Sarepta’s next-generation peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) to treat…

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September 2, 2022

WATCH: Casimir’s Duchenne Video Assessment (DVA) ARISE Study (Webinar Recording)

Casimir joined PPMD for a community webinar on Wednesday, August 31, 2022 to discuss how and why video assessments may help the Duchenne community by capturing quality of movement data in clinical trials. Casimir also…

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September 1, 2022

Pfizer Shares Update on DAYLIGHT Phase 2 Gene Therapy Trial

Pfizer has issued a community letter announcing that its Phase 2 DAYLIGHT clinical trial evaluating the company’s investigational mini-dystrophin gene therapy product (fordadistrogene movaparvovec) has opened a first trial site in Australia. Pfizer shares that…

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August 22, 2022

Santhera & ReveraGen Announce First Participant Dosed in Phase 2 Pilot Study with Vamorolone in Becker

Today Santhera and ReveraGen announced they have dosed the first participant in their Phase 2 study with Vamorolone for individuals with Becker muscular dystrophy. The small pilot study was funded through an FDA grant and…

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August 22, 2022

WATCH: Dyne Therapeutics Update on DYNE-251 (Webinar Recording)

Dyne Therapeutics joined PPMD for a community webinar on Wednesday, July 20, 2022. Dyne provided an update on the recent U.S. Food and Drug Administration (FDA) announcement lifting the DYNE-251 clinical hold and clearing the…

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August 16, 2022

2022 #PPMDConference Presentations

2022 PPMD Annual Conference Presentations JUNE 23-26, 2022 | Scottsdale, AZ Thank you to everyone who joined us for PPMD’s 2022 Annual Conference, whether you attended in person in Scottsdale, AZ, or virtually from anywhere…

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