Cong-Yue Peng

Spinal muscular atrophy in the disease-modifying therapy era: successes, limitations and future directions

Spinal Muscular Atrophy (SMA) is a rare and debilitating neurodegenerative disease characterized by the progressive loss of motor neurons in the spinal cord, leading to muscle weakness, respiratory failure, and premature mortality. The pathogenesis of SMA is highly complex and the investigation of downstream pathways a...