Early Access Program With Arimoclomol in US Patients With NPC

Inclusion Criteria: * The patient has a confirmed diagnosis of NPC (NPC1 or NPC2)\* and at least one neurological symptom. * The patient is two years of age or above. * The patient is a permanent resident of US. * If taking miglustat (Zavesca®), the patient must have been on the target dose for the past six weeks. * If the patient is a sexually active female of child-bearing potential (post-menarche), it is agreed to use highly effective contraception during the EAP and until three weeks after the last dose of arimoclomol. * Confirmed negative urine pregnancy test for sexually active female of child-bearing potential (post-menarche). * All sexually active male patients with female partners of child-bearing potential (postmenarche) agree to use a condom in addition to the birth control used by their partners during treatment and until three weeks after the last dose of arimoclomol. * If the patient has a history of seizures, the condition must be adequately controlled, i.e., the pattern of seizure activity must be stable, and the patient must be on a stable dose and regimen of antiepileptic medication during one month prior to screening. * Patient or parent/guardian must provide written informed consent to participate in EAP. * In line with Patterson et al. 2017 Exclusion Criteria: * Severe liver insufficiency. * Renal insufficiency. * The patient has a known or suspected allergy or intolerance to arimoclomol or its constituents. * The patient is pregnant, planning to be…