Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Centers (MDSRC) (P50 Clinical Trial Optional)

RFA-AR-23-001

NIAMS, NICHD, NHLBI and NINDS seek to continue this MDSRC program to advance research in the muscular dystrophies leading to improved understanding of these diseases and develop effective treatments and other strategies for reducing disease consequences.  Under this FOA, each Center application must propose a clinical research project and at least one other project, which could include basic, preclinical translational or clinical research.  The research projects should be related to a common theme and be synergistic, and should leverage the multidisciplinary and collaborative environment of this Center mechanism.  Awards are expected to contribute to the long-term goals of advancing understanding of the causes and natural history of the dystrophies, developing therapies and reducing the impact of one or more form(s) of muscular dystrophy, as well as the training, research resource sharing and patient/community outreach goals described below.  Projects should be focused only on muscular dystrophy research and may include studies of the impact of these diseases on skeletal muscle, the heart, respiratory system, sleep, smooth muscle, the central nervous system, gut or other organ systems as well as neuropsychological or neurobehavioral studies.  Areas of research can include, but are not limited to the following:

• Characterizing disease natural history or genotype/phenotype correlations
• Characterizing and/or validating molecular, biochemical, physiological, imaging or other types of biomarkers for use in therapy development or clinical trials
• Developing and/or validating clinical outcome assessment measures, including patient-reported outcome (PRO) measures, or clinical scales for use in clinical trials
• Developing innovative translational products that can really impact patient outcomes
• Identifying new muscular dystrophy causing genetic mutations  
• Identifying, characterizing and validating therapeutic targets
• Identifying and validating genetic or epigenetic modifiers of disease presentation or severity
• Engineering and characterizing new cell or animal models of disease to advance studies of pathophysiology or preclinical translation
• Screening, optimizing and preclinical testing of candidate therapeutics
• Activities leading to investigational new drug (IND) or investigational device exemption (IDE) applications to the FDA
• Developing and/or validating epidemiological, behavioral or health outcomes to assess patient- and family-centered outcomes with the eventual goal of reducing overall consequences of disease
• Optimizing symptom treatment such as respiratory support, nutritional support, and physical therapy to improve quality of life and decrease associated morbidity
• Conducting early-stage clinical trials (proof of concept trials, safety/tolerability, dose ranging studies, etc.  However, phase 3/registration trials are not permitted.)

Applicants may request up to $1,000,000 direct costs/year (exclusive of facilities and administrative costs of subcontractors with collaborating organizations).

30 days prior to the application due date

September 30, 2022

Emily Carifi, Ph.D., National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), Telephone: 301-596-0665, Email: emily.carifi@nih.gov