Last week Sheryl Sandberg -- CEO of Facebook, billionaire, best-selling author and, according to Forbes, the 7th most powerful woman in the world -- posted…
SUMMER 2016 SNAPSHOTS al fresco dinner with Hayden "stand up" paddleboarding with Wilson firepit with Jackson and the ladies There have been some heart-wrenching delays…
Earlier this week at the annual PPMD Connect Conference in Orlando, Akashi Therapeutics CMO Dr. Diana Escolar presented an update on the HT-100 program. "We…
While we aggressively advocate for the approval of eteplirsen, we remain active on other medical research fronts. It's important to remember that eteplirsen helps 13%…
A boy with Duchenne at Monday's FDA hearing on eteplirsen. On Monday, the FDA convened an advisory committee meeting to review eteplirsen, a precision medicine…
You probably already know that Charley's Fund is supporting the development of several new medicines to slow or halt Duchenne's deadly progression. Our goal is…
For several years now, I have been advocating for the advancement and approval of a new drug called eteplirsen being developed by Sarepta Therapeutics. My…