How does Niemann-Pick type C disease impact patients?

Niemann-Pick type C disease (NP-C) has a huge impact on the lives of patients, as well as their families. The progressive nature of NP-C means that children perform poorly at school and find it difficult to join in with recreational and social activities. Everyday activities for adult patients, including the ability to work or drive, become severely restricted. Patients inevitably lose their independence and have to rely heavily on those around them.

Coping with a severe, chronic disease such as NP-C presents challenges for parents and/or family members. The varied and wide ranging symptoms mean that care givers must liaise with many different medical specialists as well as get to grips with administering multiple and often complex drug treatments, in addition to other therapies such as physical therapy.

Looking after a family member with NP-C requires a considerable amount of time and energy, which although extremely rewarding, can at times be all-consuming and over-whelming. Consequently, it is essential to seek help and support. Patient associations (click for further information) are an important resource, providing access to information and counseling services. They also provide the opportunity to talk to and meet other people living with NP-C as well as to share experiences and concerns. In addition, specialist medical centers supported by highly trained medical staff are starting to be set up in some countries to provide all aspects of NP-C care.