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Clinical Trials and Drug Approval

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.

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.

Expanding the Pipeline: Industry Updates on Drug Development

Dr. Laury Mignon, from Ionis Pharmaceuticals, and Dr. Joseph Horrigan from AMO Pharma, report on the status of drug development efforts for DM patients. Jeremy Kelly and Dr. John Porter of the Myotonic Dystrophy Foundation introduce the session with a short overview on MDF's efforts to engage industry in DM therapy development. From the 2017 MDF Annual Conference.

MDF Research Update: What's Been Accomplished, What's Next

Dr. John Porter, PhD, Chief Science Officer at the Myotonic Dystrophy Foundation, reports out on the impact achieved and work carried out to date for the three year, $5M MDF 3.0 initaitive to accelerate drug development for DM, and what MDF is planning for our next drug development acceleration initiative, MDF 4.0. From the 2017 MDF Annual Conference.

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.

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.

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.

The Clinical Trial: Real-Life Experiences (2013 MDF Annual Conference)

Pat Furlong, CEO of Parent Project Muscular Dystrophy, will share the experiences of Duchenne community members who have participated in clinical trials.

(View a PDF of Ms. Furlong's presentation)

The U.S. Drug Testing & Approval Process (2013 MDF Annual Conference)

Dr. Doug Kerr, M.D., Ph.D., of Biogen-Idec, describes the U.S. process for taking a drug from testing to approval.

(View a PDF of Dr. Kerr's presentation)

Challenges of DM

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

Partners

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