The PPMD Every Single [One] Tour had an amazing turnout last weekend in Seattle, WA! Nearly 100 parents, patients and professionals gathered at Seattle Children’s Hospital to talk and learn about research, care, therapy, and clinical…

Summit Therapeutics announced today that it will proceed with the planned extension phase of PhaseOut DMD, a Phase 2 clinical trial evaluating the utrophin modulator ezutromid, subject to regulatory approval.

This follows an interim review of the safety and tolerability data from the ongoing trial by an independent Data…

Health insurance is a contract between you and an insurance company. Health insurance helps pay your medical bills when you need care.



There are two major types of health insurance:

• Private Health Insurance: Families and individuals obtain insurance privately, through an employer or group sponsor, or privately…

Today ReveraGen BioPharma Inc, announced that the FDA has granted Fast Track designation for vamorolone (VBP15) for the treatment of Duchenne.  This designation can speed the review of efficacy and safety data for vamorolone in people with Duchenne, potentially leading to more rapid regulatory approval. PPMD is pleased to learn this news and happy to have provided…

Today, PTC Therapeutics provided the following update to the Duchenne community regarding their acquisition of Emflaza™ (deflazacort) from Marathon Pharmaceuticals. We appreciate PTC keeping our community updated during this transition.

March 24, 2017

Dear Duchenne…

PPMD is pleased to learn that the FDA has completed its review and concluded that Akashi Therapeutics may resume clinical development of HT-100 (delayed-release halofuginone) in people with Duchenne (click here to read Akashi's press release). Preventing fibrosis is an important target and an essential piece of a combination of therapies that will be…

Today we learned that PTC Therapeutics has entered into an asset purchase agreement with Marathon Pharmaceuticals, LLC …

PPMD is pleased to learn that the FDA has acknowledged the filing over protest of PTC's New…