Muscular Dystrophy: Pathogenesis and Therapies (R01)
Funding Contact(s): John Porter, Ph.D.
Funding Categories: Channels, Synapses and Circuits
Brief Description:
A principal goal of this initiative is to promote research that will lead to better treatment for all muscular dystrophies.
The NIAMS, NINDS, NICHD and NHLBI encourage investigator-initiated research grant applications to study therapeutic and pathogenic
approaches. Special emphasis is on areas discussed in the MDCC Research and Education Plan and the reports of recent meetings
held at the NIH on DMD and FSHD referenced above. Important research priorities include studies on gene and stem cell therapies,
pharmacological approaches to treatment, and clarification of the role of inflammatory mechanisms. Topics for research projects
can include screening and/or diagnosis, understanding disease mechanisms, treatment strategies, and improving quality of life,
and can include studies in appropriate animal models or preclinical or clinical studies in patients. Program staff must be
contacted prior to submitting a proposal that includes a clinical trial or interventional study in humans.
Investigators with diverse scientific interests are invited to apply their expertise in basic, translational, or clinical
research to enhance our understanding of the pathogenesis and treatment of the muscular dystrophies. Applicants are encouraged
to develop innovative and novel approaches for studying and treating these diseases. Examples that illustrate possible areas
of research are presented below. They are intended only to provide a broad direction for research and should be considered
illustrative and not restrictive.