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Research Highlights

DM - Searching for Breakthroughs in Research

Dr. Charles Thornton describes the remarkable advances achieved in myotonic dystrophy research and the implications of that progress for the state of research funding overall.

DM & Anticipation, Part 1

Dr. Darren Monckton describes anticipation in myotonic dystrophy, the process by which the disease increases in severity as it is passed from generation to generation.


Watch Part 2 of this series

The Patient Registry-A Critical Research Tool

Dr. John Day describes the importance of the patient registry in moving research forward.

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.

2015 MDF Annual Conference: Early Stage Therapies - Drugs Under Development

Representatives from various biotech and pharmaceutical companies around the world discuss early stage therapies currently under development.

2015 MDF Annual Conference: DMPKrx Phase I/II Clinical Trial Update

Dr. Laurence Mignon, PhD, Director of Clinical Development at Isis Pharmaceuticals, gives an update on the status of the DMPKrx Phase I/II trial.

2015 MDF Annual Conference: Accelerating Research

Dr. Sharon Hesterlee, PhD, Chief Science Officer at MDF, discusses the Foundation's multi-year vision to accelerate DM research.

2015 MDF Annual Conference: Funding the Next Generation - MDF Fellows Report Out

MDF Fellows report on the current status of their research findings. Featuring: Dr. Ranjan Batra, PhD, University of California San Diego; Dr. Viachaslau Bernat, PhD, Scripps Research Institute Florida; Dr. Melissa Hinman, PhD, University of Oregon; and Dr. Ravi Kanth Rao Saini, PhD, Scripps Research Institute La Jolla.

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