Our Work

The INPDA’s overall mission is to assist, connect and support families affected by Niemann-Pick disease worldwide.  To achieve this, our strategy focuses on three key areas: the provision of mutual support between patient groups; the development of information; the stimulation of relevant research.

Collectively, INPDA members represent the majority of known NPD patients across world. Our network brings people together, sharing information and ideas with the aim of accelerating progress in the field of Niemann-Pick diseases. To achieve our aims and objectives, we encourage effective collaboration and the sharing of ideas across patient groups, clinical and scientific academia and the pharmaceutical industry. We provide a forum that enables networking on a global scale, reducing overlap and maximising resources.

INPDA representatives are regarded as experts in their field; they are regularly consulted regarding clinical research and trials, are involved in high level advisory boards and are invited to speak at international events and conferences.

Since its beginning in 2009 the INPDA has strived to provide a forum for patient groups and professionals working in the field of NPD to ensure that no family diagnosed with NPD should face the future alone. To do this we focus on three main areas; Providing mutual support, developing information, and stimulating research.

Establish, maintain and develop an international NPD network to enable the sharing of knowledge, resources and skills between patient groups.

Encourage the establishment of NPD support groups in countries where they do not exist.

Lobby for equity of treatment access.

Share ideas on funding opportunities.

Disseminate up to date and accurate information on clinical trials. See our Trial Updates section for most recent developments.

Increase awareness and improve diagnosis of NPC. See our Think Again, Think NPC page for more information on the campaign.

Encourage co-operative working with pharmaceutical companies and other stakeholders.

Encourage collaborative approaches. The Loire Valley Meeting is an event held by the INPDA every two years bringing researchers together from across the world to openly discuss progress and identify research priorities and potential collaborations. The aim is to accelerate progress towards future NPC treatments.

Establish an international registry of NPD patients. Find out more about the INPDR, here.