How is Niemann-Pick type C disease managed?
Recently significant advances have been made in the understanding of Niemann-Pick type C disease (NP-C). Although there is currently no cure for NPC, a new treatment and those still under development, may significantly improve the outlook for patients and offer hope for the future. Until recently, management strategies for the disease included general therapies that are used to reduce symptoms but do not influence the outcome of the disease. Symptoms that can be treated with general therapies include, but are not limited to:1 Sudden muscular weakness/collapse brought on by laughter (gelastic cataplexy) – can be treated by using tricyclic antidepressants or CNS stimulants Sustained muscle movements (dystonia) – can be treated by using anti-cholinergic drugs Movement restrictions – can be treated by using physical therapy Seizures – can be controlled by using anti-epileptic drugs Feeding problems resulting from difficulty in swallowing – can be managed with a feeding tube In January 2009
