Velmanase alfa approved for treatment of non-central nervous system manifestations of alpha-mannosidosis: A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG)

Genet Med Open. 2024 Apr 5:2:101832. doi: 10.1016/j.gimo.2024.101832. eCollection 2024.
No abstract available

Keywords: Alpha-mannosidosis; Enzyme replacement therapy; Lysosomal disorder; Velmanase alfa.