• Regulatory NewsRegulatory News

    FDA Releases Highly Anticipated Guidance on Duchenne Muscular Dystrophy

    A new draft guidance document released today by the US Food and Drug Administration (FDA) aims to clarify the development process for new drugs intended to treat Duchenne Muscular Dystrophy (DMD), a progressive, fatal disease for which there is currently no cure. Background DMD is a rare disease almost exclusively affecting young men. Due to a genetic defect, the body does not produce dystrophin, which ensures the proper function of muscle tissue. Over time, this resul...
  • Feature ArticlesFeature Articles

    Filling a Regulatory Void: Patient Advocates Submit Guidance for Duchenne Muscular Dystrophy

    Muscular dystrophy (MD)––a group of diseases that cause progressive muscle weakness due to defects in muscle proteins ––has a range of complex features that make treatments elusive. MD exists in many forms (Duchenne MD is the most common); animal models do not accurately reflect human disease; and it is difficult to define and measure the progression of this slow disease. Because MD can be life-threatening, new disease-modifying (serving to slow or halt progression) an...