Neuromuscular Disorders

Preclinical Research

Our basic research focuses on stem cell therapies and "disease-in-a-dish" models for drug development. These research projects allow us to study new ways to protect damaged motor neurons in the living tissues of individual patients.

Stem Cells

Neuromuscular researchers generate stem cells that make a protein known as glial cell line derived neurotropic factor (GDNF). We believe GDNF can protect damaged motor neurons in amyotrophic lateral sclerosis (ALS).

Disease-in-a-Dish Model for Drug Development

Working with the Regenerative Medicine Institute Brain Program, we obtain skin fibroblasts from patients with various neuromuscular diseases, convert them into induced pluripotent stem cells and then into nerve or muscle cells. These new cells allow us to study living tissues from individual patients.

Clinical Research

Our varied clinical research projects involve benefits of Diaphragm Pacing System and other possible treatments for ALS and the establishment of a repository of tissue and DNA samples to be used for future research into various neuromuscular and neurologic conditions. Those trials are:

  • Accurate Test of Limb Isometric Strength (ATLIS) in ALS
  • Genetics Repository of Neurologic and Neuromuscular Disorders
  • Natural History of CMT
  • Trial of Resistance and Endurance Exercise in ALS