You don’t have to figure this out alone
Not a real patient.
When symptoms don’t add up, it can feel overwhelming. But families don’t have to navigate Niemann-Pick type C, or NPC, alone.1,2
Managing NPC requires specialized care1,2
A healthcare provider experienced in diagnosing and managing NPC can bring clarity and support further evaluation. With the right healthcare providers, you can better understand treatment options and make confident decisions about your child’s care.
Healthcare providers experienced in diagnosing and managing NPC include1:
- Pediatric neurologists
- Metabolic-disease specialists
- Medical geneticists/genetic counselors
- Neuro-ophthalmologists
- Anesthesiologists
- Nutritionist/
gastroenterologists - Neuropsychologists
- Speech and language therapists
Find care from an NPC-experienced center
Seeing a healthcare provider experienced in diagnosing and managing NPC can help families move forward sooner. Use our Specialist Finder tool to search for healthcare providers and care centers near you.1
Not a real patient.
Find support and community
Several organizations provide support, education, advocacy, and community resources to children with NPC and their families.
National Niemann-Pick Disease Foundation
Ara Parseghian Medical Research Fund
Firefly Fund
Treatment options for NPC are expanding1,3
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 older1,4-6
Levacetylleucine in patients weighing ≥15 kg (33 lbs)7
*Miglustat is not approved for NPC in the United States but may be prescribed off-label.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. Staying informed supports families and care teams in making treatment decisions together.3
Could this be NPC?
Use this symptom checklist to help understand whether what you’re seeing could be signs of NPC.
This checklist is meant to help you understand possible symptoms and should not replace guidance from a healthcare provider. It should be used for children experiencing neurological symptoms before 6 years of age.
Which neurological signs have you noticed?
Select any symptoms that your child is experiencing now or has experienced in the past.1,2
Have you noticed any of these early signs?
Some children with NPC experience these signs after birth. Select any that your child has now or has experienced in the past.1
Is there a known family history of NPC in a biological relative?
Confirm if anyone in your family has been diagnosed with NPC.
RESULTS
It may be time to start a conversation.
Based on the signs and symptoms you selected, consider talking with your child's healthcare provider about genetic testing.
NEXT STEPS
Be prepared for the conversation
Use this guide to help prepare for a conversation with your child's healthcare provider about genetic testing.
Find experienced care
A healthcare provider experienced in treating NPC can help guide next steps, answer questions, and support your child’s care.
This checklist is meant to help you understand possible symptoms and should not replace guidance from a healthcare provider. It should be used for children experiencing neurological symptoms before 6 years of age.
NEXT STEPS
Be prepared for the conversation
Use this guide to help prepare for a conversation with your child's healthcare provider about genetic testing.
Find experienced care
A healthcare provider experienced in treating NPC can help guide next steps, answer questions, and support your child’s care.
RESULTS
If you still have concerns, don’t stop asking questions.
The information you shared doesn’t necessarily point to NPC based on this checklist alone. But you still know your child best. If you’re concerned about your child’s symptoms or how they’re changing over time, continue the conversation with your child’s healthcare provider or an NPC specialist.
NEXT STEPS
Find experienced care
A healthcare provider experienced in treating NPC can help guide next steps, answer questions, and support your child’s care.
Be prepared for the conversation
Use this guide to help prepare for a conversation with your child's healthcare provider about genetic testing.
This checklist is meant to help you understand possible symptoms and should not replace guidance from a healthcare provider. It should be used for children experiencing neurological symptoms before 6 years of age.
NEXT STEPS
Find experienced care
A healthcare provider experienced in treating NPC can help guide next steps, answer questions, and support your child’s care.
Be prepared for the conversation
Use this guide to help prepare for a conversation with your child's healthcare provider about genetic testing.
RESULTS
It may be time to start a conversation.
Based on the signs and symptoms you selected, consider talking with your child’s healthcare provider about genetic testing.
NEXT STEPS
Find experienced care
A healthcare provider experienced in treating NPC can help guide next steps, answer questions, and support your child’s care.
Find a specialist
Be prepared for the conversation
Use this guide to help prepare a conversation with your child’s healthcare provider about genetic testing.
Access conversation starter
This checklist is meant to help you understand possible symptoms and should not replace guidance from a healthcare provider. It should be used for children experiencing neurological symptoms before 6 years of age.
©2026 Beren Therapeutics P.B.C. All rights reserved. Beren and Mandos are trademarks of Beren Therapeutics P.B.C. DSA-1620-US-EN-v1 07/2026
RESULTS
If you still have concerns, don’t stop asking questions.
The information you shared doesn’t necessarily point to NPC based on this checklist alone. But you still know your child best. If you’re concerned about your child’s symptoms or how they’re changing over time, continue the conversation with your child’s healthcare provider or an NPC specialist.
NEXT STEPS
Find experienced care
A healthcare provider experienced in treating NPC can help guide next steps, answer questions, and support your child’s care.
Find a specialist
Be prepared for the conversation
Use this guide to help prepare for conversations with your child’s healthcare provider about genetic testing.
Access conversation starter
This checklist is meant to help you understand possible symptoms and should not replace guidance from a healthcare provider. It should be used for children experiencing neurological symptoms before 6 years of age.
©2026 Beren Therapeutics P.B.C. All rights reserved. Beren and Mandos are trademarks of Beren Therapeutics P.B.C. DSA-1620-US-EN-v1 07/2026
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. Patterson MC, Lloyd-Price L, Guldberg C, et al. Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale. Orphanet J Rare Dis. 2021;16(1):79. doi:10.1186/s13023-021-01719-2 3. 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 4. Miplyffa. Prescribing information. Zevra Therapeutics, Inc; 2025. 5. Zavesca. Prescribing information. Actelion Pharmaceuticals US, Inc; 2022. 6. 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 7. Aqneursa. Prescribing information. IntraBio Inc; 2024.
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