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Managing NPC requires specialized care

Not a real patient.

Niemann-Pick disease, type C (NPC), is a rare condition that often requires coordination across multiple specialties.1

NPC care takes a team1

A multidisciplinary team can provide the most comprehensive care that patients with NPC need.1

A multidisciplinary care team may include:

  • Pediatric neurologists
  • Metabolic disease specialists
  • Medical geneticists/genetic counselors
  • Neuro-ophthalmologists
  • Nutritionists/gastroenterologists
  • Neuropsychologists
  • Speech and language therapists
  • Anesthesiologists

Refer to an
NPC-experienced center

Use the Specialist Finder to locate clinicians with experience treating NPC.

Find a specialist

Referring patients to an NPC-experienced center can support an earlier diagnosis and ongoing management.1

Treatment options for NPC are expanding1,2

Approved treatment options

There are approved treatment options available for neurological symptoms of NPC, including:

Arimoclomol in combination with miglustat* in patients 2 years of age and older1,3-5

Levacetylleucine in patients weighing 
≥15 kg (33 lbs)6

*Miglustat is not approved as a monotherapy for NPC in the United States but may be prescribed off-label.1,3-5

NEW THERAPIES BEING STUDIED

Researchers are also studying new treatments that work differently to address NPC. Other therapies are currently in development in ongoing clinical trials.1

Treatment options continue to grow as we learn more about NPC.1 Staying informed supports families and care teams in making treatment decisions together.

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ORDER GENETIC TESTING AT NO COST

Beren will provide patients meeting certain eligibility criteria with sponsored whole genome sequencing and familial variant testing.

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References: 1. Hiwot T, Porter FD, Bremova-Ertl T, et al. 2025 Consensus Clinical Management Guidelines for Niemann-Pick Disease Type C. J Inherit Metab Dis. 2026;49(3):e70185. doi:10.1002/jimd.70185 2. Berry-Kravis E. Niemann-Pick disease, type C: diagnosis, management and disease-targeted therapies in development. Semin Pediatr Neurol. 2021;37:100879. doi:10.1016/j.spen.2021.100879 3. Miplyffa. Prescribing information. Zevra Therapeutics, Inc; 2025. 4. Zavesca. Prescribing information. Actelion Pharmaceuticals US, Inc; 2022. 5. Burton BK, Ellis AG, Orr B, et al. Estimating the prevalence of Niemann-Pick disease type C (NPC) in the United States. Mol Genet Metab. 2021;134(1-2):182-187. doi:10.1016/j.ymgme.2021.06.011 6. Aqneursa. Prescribing information. IntraBio Inc; 2024.

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