Support and resources

AQNEURSA Cares program

The AQNEURSA Cares program can help eligible patients access treatment. It is the single point of contact for access to AQNEURSA, and the team is there to help you or someone you care for get started once prescribed.

Logo for the AQNEURSA™ Cares program, providing patients and caregivers with patient support services

Get to know the program by downloading the brochure, and work with the prescribing healthcare provider to complete the starter form.

Helpful resources

Review these useful resources that can help with understanding and getting started on AQNEURSA.

Niemann-Pick disease type C (NPC) organizations

Learn more about the disorder and connect with the NPC community.

IntraBio is not responsible for the content or services of these organizations. Inclusion of these links on this website is not intended as an endorsement of the organizations or the services offered.

Helpful tip

The NPC community is not just there for people with NPC, it’s also there for families and those who provide care.

See answers to frequently asked questions

Important Safety Information
Before taking AQNEURSA, tell your healthcare provider about all your medical conditions, including if you are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed.
  • Based on findings from animal reproduction studies, AQNEURSA may cause harm to your unborn baby when administered during pregnancy.
  • Your healthcare provider will check to make sure you are not pregnant before starting treatment with AQNEURSA.
  • Use effective birth control (contraception) during treatment with AQNEURSA and for 7 days after stopping treatment to avoid pregnancy.
  • If you become pregnant while taking AQNEURSA, inform your healthcare provider immediately to discuss potential risks and alternative treatments.
  • It is not known if AQNEURSA or its metabolites pass into human or animal milk or effect breastmilk production.

Tell your healthcare provider about all the medications you take and if you get any new medications, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Other medicines may cause side effects or affect how AQNEURSA works when used together.

  • Tell your healthcare provider if you are taking certain medications called P-gp substrates, N-acetyl-DL-leucine or N-acetyl-D-leucine. If you are not sure if you are taking these medicines, talk to your healthcare provider or pharmacist.

The most common side effects of AQNEURSA in people treated for Niemann-Pick disease type C include abdominal pain, dysphagia, upper respiratory tract infections and vomiting.

The most common side effects of AQNEURSA in people treated for ataxia-telangiectasia include fall, skin laceration, and urinary tract infection.

These are not all the possible side effects of AQNEURSA. Call your doctor for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088 .

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Important Safety Information
Before taking AQNEURSA, tell your healthcare provider about all your medical conditions, including if you are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed.
  • Based on findings from animal reproduction studies, AQNEURSA may cause harm to your unborn baby when administered during pregnancy.
  • Your healthcare provider will check to make sure you are not pregnant before starting treatment with AQNEURSA.
  • Use effective birth control (contraception) during treatment with AQNEURSA and for 7 days after stopping treatment to avoid pregnancy.
  • If you become pregnant while taking AQNEURSA, inform your healthcare provider immediately to discuss potential risks and alternative treatments.
  • It is not known if AQNEURSA or its metabolites pass into human or animal milk or effect breastmilk production.

Tell your healthcare provider about all the medications you take and if you get any new medications, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Other medicines may cause side effects or affect how AQNEURSA works when used together.

  • Tell your healthcare provider if you are taking certain medications called P-gp substrates, N-acetyl-DL-leucine or N-acetyl-D-leucine. If you are not sure if you are taking these medicines, talk to your healthcare provider or pharmacist.

The most common side effects of AQNEURSA in people treated for Niemann-Pick disease type C include abdominal pain, dysphagia, upper respiratory tract infections and vomiting.

The most common side effects of AQNEURSA in people treated for ataxia-telangiectasia include fall, skin laceration, and urinary tract infection.

These are not all the possible side effects of AQNEURSA. Call your doctor for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088 .