Received: 18 December 2022
Accepted: 18 May 2023
First Online: 20 May 2023
: This work was approved by the Boston Children’s Hospital, Institutional Review Board. All participants provided informed consent.
: Not applicable.
: Dr. Al-Hertani is a site Principal Investigator for the Early Access Program with Arimoclomol in US Patients with NPC (NCT04316637), which is sponsored by KemPharm Denmark A/S. All other authors declare that they have no competing interests.