Blog

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…

Learn More

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…

Learn More

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…

Learn More

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.

Learn More

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…

Learn More

NS Pharma Announces First Patient Enrolled in Phase 3 Trial of Viltolarsen in Japan

NS Pharma, a wholly owned subsidiary of Nippon Shinyaku, has enrolled the first patient in a Phase 3 trial of viltolarsen in Japan. PPMD looks forward to continued updates from NS Pharma ahead of their…

Learn More

Sarepta and Dyno Therapeutics Announce Agreement to Develop Next-Generation Gene Therapy Vectors

Sarepta Therapeutics and Dyno Therapeutics announced an agreement to develop next-generation gene therapy vectors for muscle diseases. PPMD is pleased to see Sarepta’s continued investment and contribution in enhancing the future of gene therapy technologies…

Learn More

Solid Biosciences Shares Update on SGT-001 Phase I/II Clinical Hold for IGNITE DMD Program

Solid Biosciences shared today that their IGNITE DMD Phase I/II clinical trial will remain on clinical hold. Solid will continue to work with the FDA to provide further data and analyses relating to their manufacturing…

Learn More

Italfarmaco Provides Update on Advanced-Stage Clinical Development Program with Givinostat in Duchenne

Good news from Italfarmaco! As discussed on our webinar last week, an Independent Data Monitoring Committee (IDMC) has recommended the continuation of their Phase 3 Givinostat trial, determining that the trial is not futile. They…

Learn More

WATCH: Italfarmaco Provides Givinostat Update

Webinar Recording: Italfarmaco Provides Givinostat Update On April 22, 2020, Italfarmaco joined PPMD for a webinar to update the community about Italfarmaco’s development of Givinostat and the recent results of the futility analysis of the…

Learn More

Join Our Mailing List