miglustat capsules — Medica
Niemann-Pick disease type C (NPC)
Initial criteria
- Patient is age ≥ 2 years
- Diagnosis is established by a molecular genetic test showing biallelic pathogenic variants in either the NPC1 or NPC2 gene
- Medication is prescribed by or in consultation with a geneticist, endocrinologist, metabolic disorder subspecialist, or a physician who specializes in the treatment of NPC or related disorders
Approval duration
1 year