Program Description
The fellowship program in muscle disorders trains pediatric and adult neurologists in inflammatory and genetic muscle and nerve diseases in preparation for an academic career. Fellows have the opportunity to study basic disease mechanism using different model systems and develop gene and mechanism directed therapeutic approaches. Various techniques in genomic medicine and preclinical translational science can be explore (including whole exome sequencing, whole genome sequencing, RNA sequencing, gene and transcript targeting and editing, generation of inducible pluripotent stem cells and animal models.) Muscle and Nerve Disorders Fellows have the opportunity to participate in conducting therapeutic clinical trials using such pharmacological and biological approaches. A number of cohorts of patients with specific muscle and nerve disorders are followed, and a genetic outreach program is in place to identify and characterize pediatric and adult patients and their families with both new and previously known hereditary muscle and nerve diseases applying next generation genomic technologies in conjunction with extended phenotyping. The first year of this fellowship can be at the Johns Hopkins University Hospital as part of a JHU/NINDS joint neuromuscular fellowship program.
Areas of Current Research
- Congenital muscular dystrophies
- Congenital myopathies
- Inherited myopathies
- Muscular dystrophies
- Inherited disorders of the peripheral nervous system and motor neuron
- PIEZO2 deficiency syndrome
- Early onset spastic paraplegia
- Gene and RNA directed therapeutic approaches
- Muscle regeneration
- Autoimmune muscle disease, including dermatomyositis, polymyositis, and statin-triggered necrotizing myopathy.
- Inclusion Body Myositis
Application Information
Apply to this program through the NIH Graduate Medical Education Application System and check out the NINDS intramural website for more information.