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-Reference-Cited by-同舟云学术

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

Published:2023-09 Issue: Volume:36 Page:100988
ISSN:2214-4269
Container-title:Molecular Genetics and Metabolism Reports
language:en
Short-container-title:Molecular Genetics and Metabolism Reports

Author:

Sharma Reena,Hastings Caroline,Staretz-Chacham Orna,Raiman Julian,Paucar Martin,Spiegel Ronen,Murray Bryan,Hurst Bryan,Liu Benny,Kjems Lise,Hrynkow Sharon

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology

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