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Digital Academy: Clinical Trials and Drug Approval

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Accelerating The Search For Therapies: What's Happening, What's Next (2018 MDF Annual Conference)

Dr. Elizabeth Ackermann reports on the results of the 3-year MDF and Wyck Foundation-funded drug development acceleration effort, MDF 3.0, and introduces MDF’s new initiative, MDF 4.0, including the $1M gene editing project, the Myotonic Dystrophy Clinical Research Network study and other aggressive therapy development programs.

5/15/19 video
Locana - Industry Updates On Drug Development (2018 MDF Annual Conference)

Ranjan Batra, PhD, Locana, provides an update on their drug development efforts in the DM field. 

5/16/19 video
Expansion Therapeutics - Industry Updates On Drug Development (2018 MDF Annual Conference)

Representatives from biotech and pharma companies will provide updates on their drug development efforts in the DM field. Matthew Disney, PhD, Expansion Therapeutics

5/15/19 video
AMO Pharma Ltd. - Industry Updates On Drug Development (2018 MDF Annual Conference)

Joseph P. Horrigan, MD, from AMO Pharma Ltd. provides an update on their drug development efforts in the DM field. 

5/15/19 webinar
Ionis Pharmaceuticals - Industry Updates On Drug Development (2018 MDF Annual Conference)

Laurence Mignon, PhD, IONIS Pharmaceuticals provides an update on their drug development efforts in the DM field, 

5/16/19 video
Bringing the Patient Voice to CNS Targeting Drug Development in DM

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.

5/15/19 video
Bringing the Patient Voice to CNS Targeting Drug Development 2: Industry Observations and Feedback

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.

5/15/19 video
Patient-Focused Drug Development Meeting, Part 1 (2016 MDF Annual Conference)

Watch Patient-Focused Drug Development Meeting, Part 1, a video from the 2016 MDF Annual Conference.

7/11/18 video
Patient-Focused Drug Development Meeting, Part 2 (2016 MDF Annual Conference)

Watch Patient-Focused Drug Development Meeting, Part 2, a video from the 2016 MDF Annual Conference.

10/26/18 video
FDA 101

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.

4/16/18 video

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