Arimoclomol in adults with NPC in a real-world setting: Long-term data from an expanded access program in the USA April 15, 2024
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
Cardiovascular (CV) Safety and Pharmacokinetics (PK) of Serdexmethylphenidate (SDX), a Prodrug of d-Methylphenidate (d-MPH) Compared to Ritalin and Ritalin LA in a Single-Dose Crossover Study in Healthy Volunteers September 8, 2023
Serdexmethylphenidate/dexmethylphenidate effects on sleep in children with attention-deficit/hyperactivity disorder July 3, 2023
Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort study
Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment March 1, 2023
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
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