Cargando…
Long-term administration of intravenous Trappsol® Cyclo™ (HP-β-CD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II trial
BACKGROUND: Niemann-Pick disease type C (NPC) is a rare, fatal, pan-ethnic, autosomal recessive lysosomal storage disease characterized by progressive major organ failure and neurodegeneration. Preclinical studies confirmed a critical role of systemically administered hydroxypropyl-β-cyclodextrin (H...
Autores principales: | Sharma, Reena, Hastings, Caroline, Staretz-Chacham, Orna, Raiman, Julian, Paucar, Martin, Spiegel, Ronen, Murray, Bryan, Hurst, Bryan, Liu, Benny, Kjems, Lise, Hrynkow, Sharon |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2023
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10475848/ https://www.ncbi.nlm.nih.gov/pubmed/37670901 http://dx.doi.org/10.1016/j.ymgmr.2023.100988 |
Ejemplares similares
-
Elevated Alpha-Fetoprotein in Infantile-Onset Niemann-Pick Type C Disease with Liver Involvement
por: Kraus, Dror, et al.
Publicado: (2022) -
Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis
por: Hastings, Caroline, et al.
Publicado: (2019) -
Clinical disease characteristics of patients with Niemann-Pick Disease Type C: findings from the International Niemann-Pick Disease Registry (INPDR)
por: Bolton, Shaun C., et al.
Publicado: (2022) -
A Case Series on Genotype and Outcome of Liver Transplantation in Children with Niemann-Pick Disease Type C
por: Modin, Line, et al.
Publicado: (2021) -
Niemann-Pick disease type C
por: Vanier, Marie T
Publicado: (2010)