Atrium Health has been represented at the MDA Virtual Clinical & Scientific Conference.

Groundbreaking neuromuscular research – taking place right here in Charlotte, NC – is advancing muscular dystrophy treatment around the world. Through research being conducted within the McColl-Lockwood Laboratory for Muscular Dystrophy Research at Atrium Health, there may soon be a treatment for patients with limb-girdle muscular dystrophy type 2I (LGMD2I), called BBP-418.

BBP-418, also known as ribitol, is an oral treatment for patients diagnosed with LGMD. The potential therapy was initially developed by Qi Long Lu, MD, PhD, who is the director of the McColl-Lockwood Laboratory for Muscular Dystrophy Research at Atrium Health, as well as the scientific co-founder of ML Bio Solutions the biotechnology company helping to manufacture the treatment. This treatment may enhance, and partially compensate for, diminished muscle function caused by the genetic driver of the disease. Dr. Lu recently presented his research at the prestigious Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference, which took place March 15-18, 2021.

New treatment has been made possible by philanthropy.

The McColl-Lockwood Laboratory for Muscular Dystrophy Research is part of the Neuromuscular/ALS-MDA Center at Atrium Health, which is one of the most comprehensive ALS and muscular dystrophy facilities in the United States, and the Southeast’s major comprehensive facility for the study and treatment of degenerative neuromuscular diseases. The Laboratory is funded with support from the Carolinas Muscular Dystrophy Research Endowment, which was established by the McColl and Lockwood families in 2003. Through their generous gifts and the support of hundreds of community members, Atrium Health Foundation was able to recruit Dr. Lu from the University of London, England, who has assembled an international research team of scientists and technicians to conduct advanced research using multiple cell and gene therapies, gene correction, and drug screening.

Jane & Luther Lockwood, Jane & Hugh McColl, Jane Lockwood, and Thomas Lockwood pictured at the 2017 “Jeans, Genes and Geniuses” fundraiser.

For the McColl and Lockwood families, the mission to advance muscular dystrophy research is personal as their daughter Jane B. Lockwood was diagnosed with LGMD2I at five-years-old. “After taking Jane to see the world’s leading authority on LGMD we realized that we were receiving a very high quality of care at Atrium Health, so there was no reason to go elsewhere,” said Luther Lockwood, II, Jane’s father. “The high quality of care gave our family the confidence to start funding research at Atrium Health.” In addition to his family’s support of muscular dystrophy research at Atrium Health, Luther Lockwood is the chairman of ML Bio Solutions (an affiliate of BridgeBio Pharma) and serves on the board of directors at Atrium Health Foundation.