We want to share with you the video of the late Ricky Tsang who passed away recently after suffering with Ducenne Muscular Dystrophy (DMD) for over 30 years.  This is a You Tube video that Ricky did to Janet Woodcock, FDA director of the Center for Drug Evaluation and Research, concerning accelerated approval for Eteplirsen, a new drug that treats DMD, after the FDA Advisory Committee voted not to accelerate approval. 

While this video is heartbreaking, it is also an education about rare diseases and how rare disease patients suffer.  Ricky discusses the devastation caused by DMD which causes its victims to progressively lose their muscular abilities and results in life-threatening heart and respiratory conditions. Most DMD patients die in their 20's or 30's.   Here is a link to Ricky's video https://www.youtube.com/watch?v=NUjJEHmkEUg

Etepliren is made by Sarepta Therapeutics in Cambridge, Massachusetts. The Lerman Law Firm is proud to join with thousands of rare disease patients and advocates in aggressively promoting both the use of orphan drugs and the plight of rare disease patients who need those drugs NOW.  By working with Congress, the NIH, the FDA, and the pharma and biotech industry, we can accelerate the time to market of life-saving and life-changing drugs such as Eteplirsen. 

Candace Lerman, who is the Lerman Law Firm's Director of Advocacy for rare disease patients and our liaison with the biotech and pharma industry, is also a rare disease patient who used an Orphan drug to put her disease into remission.  While Candace still has multiple other health issues, her experience has convinced us that all patients deserve that opportunity.  For more information about Candace Lerman and our legislative advocacy on behalf of rare disease patients and the promotion of Orphan drugs see her blog at RareCandace.com.

We thank Director Woodcock for her accelerated approval of Eteplirsen and we look forward to similar approvals for other Orphan drugs in the pipeline.