Nonprofit praises pharma company tackling a devastating childhood disease

Recent talk of mega-mergers and quarterly results may have fixed our focus firmly on the financials, but this week one story brought our attention back to where it belongs – the patients.

We reported that the US Food and Drug Administration outlined an accelerated approval pathway for drisapersen from Dutch biotech firm Prosensa (Nasdaq: RNA) – a potential treatment for the devastating childhood disease of Duchenne muscular dystrophy. Prosena will file a New Drug Application with the FDA later this year, bringing hope to thousands of families. Currently, there is no treatment for Duchenne, a progressive muscle-wasting disease and the most common and lethal form of muscular dystrophy. Duchenne impacts one in every 3,500 boys and nearly 20,000 boys are living with the disease in the USA. Boys with Duchenne are usually diagnosed before the age of five and are in a wheelchair by age 12; most don’t survive their mid-20s.

California-based CureDuchenne, a non-profit organization that works tirelessly to raise awareness and fund research to find a cure for Duchenne, was the first advocacy organization to provide funding to Prosensa in 2004 to help develop exon skipping to treat Duchenne. Since then, CureDuchenne has continued to work closely with Prosensa.