Blog

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

Muscle & Nerve Publishes PPMD-Led Study on Contractures in Duchenne

PPMD is pleased to announce that Muscle & Nerve has published an article summarizing the “Contractures in Duchenne and Other Neuromuscular Conditions” conference hosted by PPMD with the National Institute of Neurological Disorders and Stroke…

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

Telehealth Visits: Dos and Don’ts

Telehealth Visits: Dos and Don’t During our May 6, 2020 interactive webinar on telehealth appointments, our panelists, including physicians and a nurse from PPMD’s Certified Duchenne Care Centers, discussed their recent experience utilizing telehealth appointments with…

Learn More

WATCH: Family Friday – Sibling Relationships

Family Friday: Sibling Relationships For many of us, self-quarantine is in full swing, and adjusting to a different family structure may be overwhelming. So your PPMD family is launching Family Fridays to discuss topics that…

Learn More

New Resource: COVID-19 & Duchenne Care Imperatives (Fact Sheet)

PPMD’s Care Team has created an easy-to-use fact sheet that provides high-level takeaways for the care of individuals with Duchenne and Becker muscular dystrophy during the COVID-19 pandemic, based on information from a recently published…

Learn More

End Duchenne Tour 2020: Virtual Tour Stop in Milwaukee

End Duchenne Tour 2020: Virtual Tour Stop in Milwaukee On Saturday, April 25, we spent the morning with the Duchenne community in and around Milwaukee, WI for another End Duchenne [Remote] Tour stop. Although the…

Learn More

Join Our Mailing List