PPMD is proud to have been chosen by American Academy of Pediatrics (AAP) and the Centers for Disease Control and Prevention (CDC) to help create a new interactive tool, "Physical Developmental Delays: What to Look For" that we…

Capricor Therapeutics announced continuing enrollment of, and the treatment of the first patient with Duchenne-related cardiomyopathy with CAP-1002 in the HOPE-Duchenne Phase I/II clinical trial. This is an exciting next step in the  progress of CAP-1002, an allogeneic, cardiosphere-derived stem cell (CDC) therapy.

As you may recall, PPMD hosted a webinar with Capricor last month. Click below to watch the webinar,…

Sarepta Therapeutics received notification of a PDUFA extension for the FDA's review of eteplirsen. The PDUFA date was extended by an extension period of three months to May 26, 2016. While this…

Lilly has now completed one of the largest clinical trials in Duchenne (330 patients) with disappointing results. Today the company reports that their Phase 3 study of tadalafil failed to provide evidence for efficacy in slowing decline in walking ability in boys with Duchenne. Subsequently, the company has made the decision to discontinue the open-label extension. The full update to the community is below.

Sadly, this is one more blow for this community. We are deeply…

The UK has instituted new regulations around shipping medications out of the country.  The UK is now requiring companies to provide proof of drugs that they have exported overseas.  In order to be compliant, Masters Marketing will begin to ship deflazacort, and other drugs, using trackable service providers.  Using this tracking system, Masters will be able to track orders and reduce the number of lost shipments.    

When you order medications,…