Mini-COMET: Safety and efficacy of >= 97 weeks' avalglucosidase alfa in infantile-onset Pompe disease participants previously treated with alglucosidase alfa

Pompe disease is a rare, progressive neuromuscular disorder caused by lysosomal acid α-glucosidase (GAA) deficiency and glycogen accumulation, leading to cellular dysfunction, progressive muscle damage, and functional disabilities. Patients with infantile-onset Pompe disease (IOPD) experience life-threatening cardiac, respiratory, and motor pathology from GAA deficiency. While treatment with alglucosidase alfa has improved the survival, cardiac function, and motor development of patients with IOPD, a significant unmet clinical need remains.