2023-2022

Evaluation of the Long-Term Effect of Arimoclomol in NPC – 48 Months Data from CT-ORZY-NPC-002

October 4, 2023

Real World Data Collection in Niemann-Pick Disease Type C – Data from Expanded Access Program with Arimoclomol

Association Between NPC Severity Score Domains and Corresponding Items of the Performance-based Scale for the Assessment and Rating of Ataxia (SARA)

February 23, 2023

Evaluation of the Long-Term Effect of Arimoclomol in NPC

Dose Proportionality and Effects of Food on the Pharmacokinetics of Single-Entity Serdexmethylphenidate

January 26, 2023

Mass Balance and Metabolic Pathway Following Oral Administration of [14C]-Serdexmethylphenidate, a Novel Prodrug of d-Methylphenidate