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Digital Academy: Clinical Trials and Drug Approval

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Clinical Trials Training

The Myotonic Dystrophy Foundation 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.

4/16/18 video
Patient-Focused Drug Development Meeting, Part 1 (2016 MDF Annual Conference)

 

MDF 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 MDF Annual Conference.

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

MDF 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 MDF Annual Conference.

10/26/18 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
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.

PowerPoint Presentation

Click here for more presentations and videos from the 2018 MDF Annual Conference in Nashville, TN.

5/15/19 video
Locana - 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. 

Speaker:
Ranjan Batra, PhD, Locana

PowerPoint Presentation

Click here for more presentations and videos from the 2018 MDF Annual Conference in Nashville, TN.

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. 

Speaker:
Matthew Disney, PhD, Expansion Therapeutics

PowerPoint Presentation

Click here for more presentations and videos from the 2018 MDF Annual Conference in Nashville, TN.

 

5/15/19 video
Ionis Pharmaceuticals - 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. 

Speaker:
Laurence Mignon, PhD, IONIS Pharmaceuticals

PowerPoint Presentation

Click here for more presentations and videos from the 2018 MDF Annual Conference in Nashville, TN.

5/16/19 video
Challenges of DM

People living with myotonic dystrophy have a frank discussion about the biggest challenges facing their lives.

4/16/18 video

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