Adult receiving clinical trial treatment for Niemann-Pick type C

Is there a treatment or cure for
Niemann-Pick Type C?

There is no cure for Niemann-Pick Disease Type C (NP-C). But there is still hope - through clinical trials of new therapies, a cure is possible.

Patients benefit from palliative treatments (individual medications that will help to treat the symptoms related to the condition). Occupational therapy can be used to help with posture, speech and movement.

In 2009, one treatment was made available upon approval for the treatment of progressive neurological manifestations in adult and paediatric patients with NPC.  It had been shown to delay the progression and stabilise certain symptoms of the disease. However, this drug is not suitable for every affected individual and you are advised to discuss all medical issues with your doctor.

There are several clinical trials currently taking place investigating new therapeutic options for patients with NPC. Please see below for current updates, or contact us for more information.