Olipudase alfa enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD): sustained improvements in clinical outcomes after 6.5 years of treatment in adults

BACKGROUND: Enzyme replacement therapy with olipudase alfa, a recombinant human acid sphingomyelinase (rhASM), is indicated for non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in children and adults. An ongoing, open-label, long-term study (NCT02004704) assessed...

Descripción completa

Detalles Bibliográficos
Autores principales: Lachmann, Robin H., Diaz, George A., Wasserstein, Melissa P., Armstrong, Nicole M., Yarramaneni, Abhimanyu, Kim, Yong, Kumar, Monica
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10131350/
https://www.ncbi.nlm.nih.gov/pubmed/37098529
http://dx.doi.org/10.1186/s13023-023-02700-x

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10131350/
https://www.ncbi.nlm.nih.gov/pubmed/37098529
http://dx.doi.org/10.1186/s13023-023-02700-x

Ejemplares similares