“In the pre-clinical setting, CK-089 increased muscle force and function in animal models of a neuromuscular disease characterized by muscle weakness, atrophy and fatigue, suggesting it may have ...
Approximately 5,000 people in the U.S. develop amyotrophic lateral sclerosis (ALS) each year. On average, they survive for only two to five years after being diagnosed, according to the Centers for ...
Researchers identify a key pathway leading to neurodegeneration in early stages of ALS, hinting at the potential for short-circuiting the progression of the fatal disease if diagnosed early.
Infants with spinal muscular atrophy (SMA) who are presymptomatic and given the gene therapy Zolgensma in the first six weeks of life have better motor, respiratory, and nutritional outcomes, ...