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
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
Pharmacokinetics and Abuse Potential of Benzhydrocodone, A Novel Prodrug of Hydrocodone, After Intranasal Administration in Recreational Drug Users January 26, 2023
Oral pharmacokinetics of KP511, a prodrug of hydromorphone, relative to hydromorphone in human volunteers
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