Olipudase alfa approved for pediatric and adult patients with acid sphingomyelinase deficiency (ASMD): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG)
oleh: Monica Penon-Portmann, Sheri A. Poskanzer, Jaya Ganesh, Irene Chang
| Format: | Article |
|---|---|
| Diterbitkan: | Elsevier 2023-01-01 |
Deskripsi
Subjek
Acid sphingomyelinase deficiency; Enzyme replacement therapy; Lysosomal storage disease; Niemann-Pick disease; Olipudase alfa