March 18, 2020

Catabasis Shares Update on Edasalonexent and COVID-19

Catabasis Pharmaceuticals has shared an update responding to questions about edasalonexent and COVID-19. To date, Catabasis has not identified any safety concerns related to COVID-19, and they continue to monitor the trial for the safety…

Learn More

February 26, 2020

Catabasis Shares February 2020 Newsletter

Catabasis shares the latest edition of their community newsletter, including an update on edasalonexent, as we approach Rare Disease Day. Click here to download (PDF).

Learn More

January 21, 2020

Santhera Shares Winter 2020 Community Update

Santhera Pharmaceuticals shared a letter with the community, emphasizing that their main focus in the U.S. right now is to bring the SIDEROS clinical trial to full enrollment. Learn more about this clinical trial, including…

Learn More

January 8, 2020

Catabasis Shares January 2020 Newsletter

Catabasis shares the latest edition of their community newsletter, including information about the Phase 3 PolarisDMD trial enrolling the expected patient population and a partnership with our friends at Duchenne UK to evaluate edasalonexent in…

Learn More

December 19, 2019

Mallinckrodt Terminates BRAVE Study Investigating the Efficacy and Safety of MNK-1411 in Duchenne

PPMD is disappointed to learn that Mallinckrodt has made the decision to terminate their BRAVE Study (MNK14112096). Based on the company’s statement below, the decision appears to have been the result of low enrollment and…

Learn More

November 19, 2019

Santhera shares SIDEROS clinical trial and BreatheDMD program update

Santhera Therapeutics shared a community letter today announcing that the company has decided to suspend enrolling new participants in the BreatheDMD expanded access program (EAP) until their SIDEROS clinical trial is fully enrolled. The SIDEROS…

Learn More

November 13, 2019

Catabasis Shares November 2019 Newsletter

Catabasis shares the latest edition of their community newsletter, including information about the role of NF-kB in muscle and an update about edasalonexent in development for the treatment of Duchenne. Click here to download (PDF).

Learn More

October 22, 2019

Reveragen Biopharma Receives NIH Ethics Grant to Return Data to Participants in Vamorolone Clinical Trials

The National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH) has awarded an ethics research grant to ReveraGen BioPharma to return patient-level and aggregate data to Duchenne patients and…

Learn More

October 7, 2019

Santhera Announces Presentation by ReveraGen of Positive 18-Month Data with Vamorolone in Duchenne

Santhera Pharmaceuticals has announced the presentation of data showing continued improvement of muscle function and improved tolerability compared with corticosteroids of 18-month vamorolone treatment in Duchenne. PPMD was an earlier funder of this important research…

Learn More

October 1, 2019

Catabasis Completes Phase 3 PolarisDMD Trial Enrollment

Catabasis announced that the Phase 3 PolarisDMD trial for edasalonexent in Duchenne has completed enrollment and exceeded the target enrollment. PPMD is proud that The Duchenne Registry was able to play a role in helping…

Learn More