Biotechnology company developing gene therapy products for patients with diseases caused by single-gene defects, including rare neuromuscular, metabolic, and cardiac disorders
Biotechnology company developing gene therapy products for patients with diseases caused by single-gene defects, including rare neuromuscular, metabolic, and cardiac disorders. Its pipeline included programs for X-linked myotubular myopathy, Crigler-Najjar syndrome type 1, Pompe disease, and catecholaminergic polymorphic ventricular tachycardia.