Update on safety and efficacy results for phase I/II trial of hydroxypropyl betacyclodextrin (HPâCD) administered intravenously in patients with Niemann-Pick disease type C1

Publication date: February 2021Source: Molecular Genetics and Metabolism, Volume 132, Issue 2Author(s): Julian A.J. Raiman, Reena Sharma, Martin Paucar, Orna Staretz-Chacham, Ronen Spiegel, Alan Boyd, Caroline Hastings, Sharon H. Hrynkow
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research