Myotonic Dystrophy Foundation
1004A O’Reilly Avenue
San Francisco, CA 94129
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- Research Portfolio
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The Myotonic Dystrophy Foundation (MDF) is the world's largest patient organization focused solely on myotonic dystrophy. Our mission, "Care and a Cure," is to enhance the quality of life of people living with myotonic dystrophy (DM) and advance research focused on treatments and a cure.
MDF has recently received a significant donation which is allowing the organization to double its operating budget in this and coming years.
MDF has implemented a multi-pronged strategy to support its mission of Care and a Cure for Myotonic Dystrophy. We believe that these objectives are tightly inter-related and necessary to support of both objectives.
- Care. Strengthen capacity and infrastructure in DM clinical care to drive more accurate clinical trial design, improve capacity to evaluate drug efficacy and advance understanding of disease course.
- Research. Deepen and strengthen the research and development bench to drive more myotonic dystrophy discovery.
- Therapy Development. Expand the drug development pipeline with additional pharmaceutical partners and additional translational research and more critical data. Incentivize Investment in myotonic dystrophy via key studies, data collection, and industry partnerships.
- Targeted and immediate advocacy effort with federal agencies and legislators to support objectives 1-3.
Research Strategies for MDF include building capacity in the field for basic research and discovery and supporting the development of new therapies.
1. Building Capacity.
a. MDF maintains a fellowship program to identify and train new investigators in the field. Fellows receive training in grant writing and presentation skills and supported to travel to two major conferences in the field per year ($55,000/year for two years, RFA, 2016 in review)
b. MDF supports the development of research tools including cell lines (fibroblast lines and iPSCs, in development), mouse models (2016) and mouse standard operating procedures (2016).
2. Supporting the Development of New Therapies. Research projects are focused on the following topics, either via release of RFA or via internal projects:
a. Assay development and screening ($200,000 over one year, RFA, in review)
b. Endpoint development, ($100,000 over one year, RFA, to be released October of 2015)
c. Biomarker development ($100,000 over one year, RFA, to be released 2016)
d. Population-based prevalence study ($50,000 for six months, RFA, phase I ready to award)
e. Burden of Disease study with Optum Labs (internal project - underway)
f. Support for Clinical Research Network/Improving Natural History Data: hiring a network director (underway); and supporting 3-4 additional sites (identified by end of 2015)
g. Merging of existing registries (internal project - in development)
h. Benefit-Risk Study (internal project - complete)
i. Regulatory workshop on myotonic dystrophy, with FDA (completed)
Fiscal year 2014, ending 12/31/14
- Revenue: $727,525
- Assets: $1,642,464
- Grants: $213,178
- Gifts Received: $696,805
- Total Expenditures: $1,088,137
- Funded 17 Postdoctoral Fellows since 2010 for a total of $1,731,925 and at least three to date have gone on to tenure track faculty positions with a focus on myotonic dystrophy.
- Established a patient registry in 2012 that now has over 1400 registrants; helped recruit for phase I/II clinical trial of Isis DMPKRx and University of Rochester's natural history study.
- Held a regulatory workshop on myotonic dystrophy with six FDA speakers, five of whom stayed for the full day, and which was also attended by 75 representatives from academia, industry and government.
- Completed a pilot benefit/risk study for patient preference in myotonic dystrophy with Silicon Valley Research (preliminary results presented)
- Established a working group with committee chairs to develop consensus-based care guidelines, and held first in-person meeting.
- Established Myotonic Dystrophy Drug Development Roundtable; first meeting attended by over 50 translationally focused academic investigators, government representatives, three large pharma and multiple biotechnology companies.
MDF has a Board of Directors composed of individuals with backgrounds in business, medicine, law and science, and other professional fields, many of whom have affected family members and some of whom are affected themselves. The current Board roster is available here.
The MDF Scientific Advisory Committee (SAC) is composed of world renowned investigators with expertise in science, medicine and drug development. The current SAC roster is available here.
Ask Us About
- Facilitating patient engagement in the R&D process
- Interaction with regulatory agencies
- Partnering with industry