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

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

doi:10.2139/ssrn.4432637

2023

DOI: 10.2139/ssrn.4432637

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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

Reena Sharma

Caroline Hastings

Orna Staretz‐Chacham

et al.

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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

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