March 17, 2021

PPMD’s Certified Duchenne Care Center Program – 2020 Impact & Progress

PPMD’s Certified Duchenne Care Center (CDCC) Program was developed to make comprehensive Duchenne care and services, provided in agreement with the CDC Care Considerations, available and accessible to as many families as possible, and to strengthen clinical trial…

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March 15, 2021

Solid Reports Efficacy & Safety Data from Ongoing IGNITE DMD Clinical Trial & Resumption of Patient Dosing

Solid Biosciences has provided a safety and efficacy update on the ongoing IGNITE DMD phase I/II clinical study of SGT-001 microdystrophin gene therapy in patients with Duchenne. Upcoming Webinar: Join PPMD and Solid tomorrow, March…

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March 4, 2021

Santhera Announces Completion of First 6-Month Period of VISION-DMD Trial with Vamorolone in Duchenne

Santhera Pharmaceuticals has reached an important milestone in the VISION-DMD study of vamorolone in people with Duchenne, conducted by partner ReveraGen Biopharma Inc. The company today announced that it has completed the last visit of…

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February 25, 2021

FDA Grants Accelerated Approval to AMONDYS 45™ (Casimersen)

PPMD is excited to learn that the FDA has granted accelerated approval to AMONDYS 45™ (casimersen), a treatment of Duchenne in patients who have a confirmed mutation of the DMD gene that is amenable to…

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February 16, 2021

Save the Date for PPMD’s Annual Conference: Virtual Edition – June 23-26, 2021

PPMD’s Annual Conference – Virtual Edition June 23-26, 2021 It’s hard to believe that we are still in this pandemic. We are getting closer to safer days ahead and we want our Duchenne and Becker…

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February 5, 2021

PTC Shares Results of Study 045 of Translarna™ (Ataluren) in Patients with Nonsense Mutation Duchenne

Twenty years ago, we were first introduced to PTC Therapeutics. We watched as PTC-124 was in early studies of safety and dose finding. Over the years, as studies continued, PTC-124 was referred to as ataluren…

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February 1, 2021

Santhera Shares Community Update on Phase 2b Trial for Vamorolone

Santhera Pharmaceuticals has shared an update with the community regarding the phase 2b trial for vamorolone, which the company expects to have data from in the second quarter of 2021. The update includes contact information…

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January 21, 2021

Postponed: SRP-9001 Update – Community Webinar

Postponed: SRP-9001 Update UPDATE: Sarepta will have to postpone the community webinar scheduled for today, January 21 at 12 PM EST. PPMD is deeply disappointed as we were hopeful to discuss current data and ask…

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January 7, 2021

Sarepta Announces Top-line Results for Part 1 of Study 102 Evaluating SRP-9001 Gene Therapy

Sarepta Therapeutics today announced top-line results from Part 1 of Study SRP-9001-102 (Study 102), an ongoing, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy and tolerability of a single dose of SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin)…

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January 7, 2021

Pfizer Doses First Participant in Phase 3 Study for Duchenne Investigational Gene Therapy

More exciting news for our community! Pfizer today announced that the first participant has been dosed in the Phase 3 CIFFREO study, which will evaluate the efficacy and safety of their investigational AAV9 mini-dystrophin gene…

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