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:

  • Completion of small, critical studies that result in a simple go/no go answer on a potential therapeutic at the earliest stages of development.
  • 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.
  • Developing and supporting the infrastructure needed by the community to complete preclinical research and clinical trials.

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

  • IND-Planning Grants are awarded to academic investigators with preclinical projects that lead directly to the submission of an investigative new drug (IND) application with the FDA. These projects may include manufacturing a potential drug substance, scale-up and toxicology studies. Academic clinical trials that are not part of a drug development program are funded through MDA’s research grant program.
  • Infrastructure Grants are designed to fund the development of infrastructure — tools, techniques or services — that will be useful to the neuromuscular disease research community for the purpose of therapy development.