NEW: Neuromuscular Antibodies
available from Iowa Hybridoma Bank
NEW RESOURCE: MDA is Pleased to Announce that the Jain Foundation is Providing New Research Tools for Dysferlinopathy
NEW: NIH Clinical Collection Now Available
NEW: NHLBI Gene Therapy Resource Program (GTRP)
The Muscular Dystrophy Association's Translational
Research Program represents an innovative
and different way of funding research - one
designed to move new drug targets into the
clinic as rapidly and efficiently as possible.
This program was developed in response to the increasing number of promising therapeutic avenues for neuromuscular disease that have arisen from MDA's basic research program. Because for-profit companies are more interested in developing drugs for large disease markets, MDA recognized the need for a program to jump-start drug development for the all rare diseases in its program.
More specifically, MDA defines "Translational Research" as:
Preclinical activities leading up to a clinical trial, including
- Toxicology
- Optimization
- Formulation
- Manufacturing and Scale-up, and
- Regulatory compliance
AND Activities supporting the conduct of clinical trials, including
- Clinical trial design
- Database development
- Protocol development
- Regulatory compliance
- Data safety monitoring
The Translational Research Program directs funds to goal-driven drug development projects in four main categories:
The program is guided by the MDA Translational Research Advisory Committee (TRAC) made up of scientists, clinicians and businessmen. The TRAC meets twice a year to evaluate new therapeutic ideas and develop strategies for moving promising candidate treatments out of the laboratory and into the clinic. At its first meeting, the TRAC developed a Strategic Plan to focus the efforts of the program.
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