Deborah Miller and Salina Prasad of the US Food & Drug Administration provide an overview of the agency and discuss patient advocacy as it relates to the FDA.
Pat Furlong, CEO of Parent Project Muscular Dystrophy, will share the experiences of Duchenne community members who have participated in clinical trials.
Dr. Doug Kerr, M.D., Ph.D., of Biogen-Idec, describes the U.S. process for taking a drug from testing to approval.
People living with myotonic dystrophy have a frank discussion about the biggest challenges facing their lives.
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