Tomorrow is a big day. A special advisory committee convened by the FDA will spend all day reviewing the first Duchenne treatment up for approval in the US. By the end of the day, the “ad comm” will answer questions FDA reviewers have posed to help them decide whether to approve the treatment. Regardless of… Read more »
Monthly Archives: November 2015
The Race to Yes: Where Do We Stand Today?
July 2012 Drug company Sarepta announces that after 36 weeks of dosing, DMD participants using eteplirsen outperformed the placebo. The company had demonstrated three months earlier that eteplirsen results in the production of significant levels of dystrophin, the protein boys with Duchenne are missing. December 2012 Sarepta requests a meeting with the FDA to determine… Read more »