There is currently no cure for NP-C, and until recently, treatments focused only on managing symptoms. These therapies may be at least partially successful in treating some symptoms of NP-C, but have no impact on disease progression or long-term outcomes. Symptoms that can be treated with general therapies include, but are not limited to:

  • Seizures - can be controlled by using anti-epileptic drugs
  • Involuntary and sustained muscle contractions leading to abnormal posture, twisting and repetitive movements (dystonia) - can be treated by using anti-cholinergic drugs
  • Movement impairment - can be treated by using physical therapy
  • Feeding problems resulting from difficulty in swallowing - can be managed with a feeding tube

In January 2009, a drug (a small iminosugar) that inhibits the enzyme responsible for the first step in the synthesis of glycosphingolipids was approved in the European Union as the first treatment specifically for patients with NP-C with progressive neurological disease. In clinical studies, this drug has been shown to slow down progression of neurological symptoms.

 

US RESIDENTS

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Please note that the information contained in this section of the website is not intended for US residents. Please visit our US webpage.