There is currently no cure for Niemann-Pick diseases, although there are some effective therapies which can often help manage and slow the progression of symptoms. Any further positive development in this area hinges on research, which is why the International Niemann-Pick Disease Registry (INPDR) was established to encourage and enable research which will someday lead to effective therapeutic options.
By enabling appropriate access to anonymised data, the INPDR will encourage efficient and timely diagnosis, improve understanding of disease progression and influence patient care, whilst supporting global research efforts.
The INPDR is already updated with information from diagnostic centres and expert clinicians from around the world. With your help, the INPDR has the potential to continue to grow as an invaluable source of information, which will give an insight into the natural history of Niemann-Pick diseases, its variability amongst the patient population, and other key diagnostic and treatment details which will help to further bolster research efforts moving forward.