Turning Research into Treatments

MDA’s translational research program represents an innovative way to fund research that’s designed to move new drug targets into the clinic as rapidly and efficiently as possible.

The program was developed in response to the increasing number of promising therapeutic avenues for neuromuscular diseases that have developed from MDA's basic research program. The program focuses on key areas MDA has identified as critical weaknesses in the drug development process for rare diseases.

Key areas include:

• Supporting early-stage (preclinical and early clinical studies) high-risk studies at companies that might otherwise not enter the rare disease arena, effectively making potential therapeutics more attractive to investors.
• Supporting the preclinical work necessary to file an investigational new drug application (IND), such as manufacturing, scale-up and toxicology studies.
• Helping academic investigators complete critical preclinical research to determine if potential therapeutics developed in academia can be developed as drugs, and to enhance the value of such therapeutics to drug development companies.

MDA’s translational research program offers the following grant types:

• MDA Venture Philanthropy is MDA’s program that directly funds and supports drug development programs in industry or in academia. Proposed projects should be focused on translational aspects of therapy development (e.g., optimization, scale-up, manufacturing, toxicology testing and phase I/II clinical trials). Awards are made based on the understanding that a therapeutic product will be brought to market by the awardee either during the tenure of the project or subsequent to the award.
   
• Clinical Research Training Grants are designed to provide promising young clinicians with the research training opportunities needed to become productive clinical investigators in neuromuscular disease research. The training opportunity is intended to be compatible with the requirements of a traditional clinical fellowship in neuromuscular disease and any forthcoming requirements for certification in neuromuscular disease