- Tytuł:
- Consensus statement on enzyme replacement therapy for mucopolysaccharidosis IVA in Central and South-Eastern European countries.
- Autorzy:
- Źródło:
- Orphanet journal of rare diseases [Orphanet J Rare Dis] 2022 May 10; Vol. 17 (1), pp. 190. Date of Electronic Publication: 2022 May 10.
- Typ publikacji:
- Journal Article; Research Support, Non-U.S. Gov't
- MeSH Terms:
-
Mucopolysaccharidoses*/drug therapy
Mucopolysaccharidosis IV*/drug therapy
Amino Acid Metabolism, Inborn Errors ; Consensus ; Enzyme Replacement Therapy ; Humans ; Isovaleryl-CoA Dehydrogenase/deficiency - SCR Disease Name:
- Acidemia, isovaleric
Czasopismo naukowe