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...

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Detalles Bibliográficos
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:
Research Paper
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

Internet

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

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