Kronn D, Davison J, Broomfield A, Brassier A, et al. The Mini-COMET Clinical Trial: Safety and Efficacy of Avalglucosidase Alfa after
97 Weeks of Treatment in Children with Infantile-Onset Pompe Disease Previously
Treated with Alglucosidase Alfa. J Pediatr 2025 May 29:114664. doi: 10.1016/j.jpeds.2025.114664.
PMID: 40449831
|