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.
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.
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.
Representatives from various biotech and pharmaceutical companies around the world discuss early stage therapies currently under development. Featuring Dr. Friedrich Metzger, PhD, Head of Discover in Rare Diseases at Roche; Dr. Takako Moriguchi, PhD, Senior Scientist at Genzyme; and Michael French, Chief Executive Officer at Marina Biotech.
Dr. Laurence Mignon, PhD, Director of Clinical Development at Isis Pharmaceuticals, gives an update on the status of the DMPKrx Phase I/II trial.