Myotonic Dystrophy Foundation

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Presentation transcript:

Myotonic Dystrophy Foundation Programs Overview June 2017

CARE Toolkits & Guidelines Website Warmline Annual Conference Digital Academy Newsletters Care Guidelines Find a Doctor Tool Publications Toolkits: Anesthesia, SSA, developmentally disabled children 0-21 planning guideline Annual conference: 300+ patients, family members, industry professionals and academic researchers; focus on symptom management, research updates & community building Clinical care guidelines: first-ever for disease: for DM1 adults, DM2, congenital & childhood as well as specialty for cardiologists, pulmonologists, gastroenterologists, OTs, PTs Warmline taking live calls daily Monthly newsletters to the community (Dispatch), industry & academia (DM Research News – the only DM-only newsletter for professionals Website: comprehensive information and resources for community members, and section for professionals Much more

CURE CLINICAL CARE RESEARCH DRUG DEV ADVOCACY PAYORS & ACCESS International Clinical Care Recommendations Clinical Research Coordinator Training and Recognition Program CRC & PT Conference Travel Grants Care Landscape Analysis and SWOT NCATs, academic & industry fellowships Expansion IP-Free BAC-Transgenic CRISPR Mouse Model IP-Free iPSC Cell Line Library Mouse SOPs SAC Expansion and Development Clinical Research Network Expansion Biomarkers & FDA Qualification Endpoint Development Industry Drug Screening Grants Regulatory Advocacy International Endpoint SOPs Registry Expansion to Longitudinal Data, PMS Muscular Dystrophy Coordinating Committee Federal agency advocacy for research funding Population-Based Prevalence Study Burden of Disease Study FDA Workshop on Clinical Trials 1st Externally-Led PFDD meeting Voice of the Patient Report TIRS publication

Research & Development Reimbursement & Access IMPACT CARE & A CURE Clinical Care Improved clinical care landscape More accurate clinical trial design Improved capacity to evaluate drug efficacy Better understanding of disease course Research & Development More dynamic and growing DM research field Increased efficiency of research output More reliable research findings More populated drug development pipeline Drug Development Optimal drug review time Improved trial processes Improved clinical trial readiness More pharma investment, exploration Reduced trial risk Advocacy More industry engagement in DM drug development Enhanced case for reimbursement Influence over pricing/access Reimbursement & Access Public & private payers reimburse DM family members for approved therapies Approved therapies pricing more community-friendly