June 26, 2020

Sarepta Completes Submission of New Drug Application Seeking Approval of Casimersen (SRP-4045)

PPMD is excited to learn that Sarepta Therapeutics has completed the submission of a rolling New Drug Application (NDA) to the FDA seeking accelerated approval for casimersen (SRP-4045). Casimersen, a phosphorodiamidate morpholino oligomer (PMO), is…

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June 19, 2020

WATCH: Antisense Therapeutics Phase II Trial Final Data Results – Webinar Recording

Antisense Therapeutics recently announced that their Phase II clinical trial of ANP’s immunomodulatory therapy, ATL1102 for Duchenne in non-ambulatory patients has met its primary endpoint, confirming the safety and tolerability of ATL1102 for advancement into…

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June 15, 2020

Sarepta Announces Positive Safety and Efficacy Data from SRP-9001 Gene Therapy Trial Published

Sarepta Therapeutics today announced safety and tolerability data at one year from four Duchenne clinical trial participants who received SRP-9001 micro-dystrophin (AAVrh74.MHCK7.micro-dystrophin) have been published in JAMA Neurology. PPMD is proud to have been an…

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June 4, 2020

WATCH: Capricor’s HOPE-2 Final 12-Month Data – Webinar Recording

Capricor Therapeutics recently announced positive final 12-month results from the HOPE-2 Trial. HOPE-2 was a randomized, double-blind, placebo-controlled study focused on later-stage Duchenne patients. On June 3, 2020, Capricor joined PPMD for a webinar to…

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June 2, 2020

Santhera Announces Completion of ReveraGen’s Long-Term Extension Study with Vamorolone in Duchenne

PPMD is excited to learn that Santhera Pharmaceuticals and partner ReveraGen Biopharma Inc. have completed a long-term, open-label extension study of 24 months duration with vamorolone in patients with Duchenne. Data from this study in…

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May 28, 2020

ReveraGen to Share Individual Patient Results with Trial Participants

ReveraGen has provided a letter to participants in their vamorolone trial indicating promising results with strength and mobility of patients on drug. Additionally, thanks to a NIH grant, ReveraGen will be sharing individual results with…

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May 20, 2020

Santhera Completes Enrollment of Phase 3 SIDEROS Study with Puldysa® (Idebenone) in Duchenne

Santhera announced today that their SIDEROS study is fully enrolled, with 254 participants ages 10-34 years old. PPMD is excited to learn that this trial is enrolled, allowing the study to continue and enabling Santhera…

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May 15, 2020

Pfizer Shares New Phase 1b Results of Gene Therapy in Ambulatory Boys With Duchenne

PROGRESS! Pfizer announced updated Phase 1b clinical data on PF-06939926, their investigational gene therapy being developed to treat Duchenne. The preliminary data from 9 ambulatory boys with Duchenne, aged 6 to 12 indicate that the…

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May 14, 2020

Catabasis Shares May 2020 Newsletter

Catabasis Pharmaceuticals has shared the latest edition of their community newsletter, including updates on edasalonexent and data shared during a recent scientific poster session. Click here to download the PDF.

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May 13, 2020

Capricor Announces Positive Top-Line Final Results from HOPE-2 Study

PPMD is excited to learn that Capricor Therapeutics has announced positive top-line 12-month results of the HOPE-2 clinical trial using CAP-1002 to treat non-ambulatory patients with Duchenne. Capricor has reported that the placebo patients in…

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