Clinical Trials and Drug Approval
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Video #2 from the session: Bringing the Patient Voice to CNS Targeting Drug Development in DM. Dr. Gersham Dent, a neuroscientist working on the DM1 drug development program at Biogen, provides observations on panelists' CNS symptom experiences with regard to possible endpoint identification for future clinical trials of CNS-targeting therapies. From the 2017 MDF Annual Conference.
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MDF community members reported on the impact of myotonic dystrophy on the brain from their individual perspectives as people living with DM1, DM2 and as caregivers. Additional insights and comments were provided by members of the conference audience. The session input will be published and used to help drug developers understand the impact of DM on the brain from the patient perspective, and begin to identify clinical trial endpoints. From the 2017 MDF Annual Conference.
Myotonic community members living with DM and their caregivers present on current symptom management strategies for living with DM, and what they would like to see in clinically-meaningful therapies. Dr. Jonathan Goldsmith, M.D., FACP, head of the Office of Rare Diseases at the FDA, provided a summary of the proceedings and what the FDA took away from the patient input shared at this meeting – at the 2016 DM Patient-Focused Drug Development meeting, a component of the 2016 Myotonic Annual Conference.
Myotonic community members living with DM, and their caregivers present on the impacts of DM on daily living, in response to specific questions posed by the Food and Drug Administration, which is in charge of reviewing and approving therapies in the U.S. Dr. Janet Woodcock, M.D., who leads all drug evaluation and research at the FDA, also provided remarks – at the 2016 DM Patient-Focused Drug Development meeting, a component of the 2016 Myotonic Annual Conference.
MDF presents a training video for clinical trials participants, presented by Dr. Bruce Wentworth of Genzyme, and Jeanne Dekdebrun and Elizabeth Luebbe of the University of Rochester.
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.
People living with myotonic dystrophy have a frank discussion about the biggest challenges facing their lives.
