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Care and Support

Our main focus, above all else, is to provide unique care and support services to families affected by Niemann-Pick diseases. We originally started out as a parent support group made up of ten families affected by Niemann-Pick disease and 25 years later we stand stronger than ever.

Since 1991 we have continually developed our service so we can better serve those who need it most. Many of the individuals who work within the charity have been directly affected by Niemann-Pick in their personal lives, which adds a unity and shared-understanding of the potential struggles and subsequent help you may need.

Through the dedicated work of our Care and Support team, which consists of NPUK Clinical Nurse Specialist Laura Bell, NPUK Senior Families Advocate Elizabeth Davenport, and NPUK Project Families Officer Steve Neal, we work to ensure that our community feel well supported.

Care and Support

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Latest news

29.11.19

#ChildrenAtTheHeart

We are joining 140+ organisations in a call to all political parties to put children and young people at the heart of this General Election...

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19.11.19

Orphazyme receives Breakthrough Therapy Designation for arimoclomol in Niemann-Pick Disease Type C (NPC)

Kim Stratton, Chief Executive Officer at Orphazyme, commented, “We are delighted with FDA’s decision to grant Breakthrough Therapy Designation to arimoclomol for NPC. Arimoclomol has been shown to have a clinically meaningful effect on disease progression in NPC that is further supported by a biomarker effect indicating an effect on the biological underpinnings of the disease and a favorable safety and tolerability profile. We are committed to bringing this product to patients as soon as possible. Breakthrough Therapy Designation is designed to expedite the development and review of products for serious diseases with the direct involvement of senior staff and we look forward to working closely with the FDA to further advance arimoclomol. Our preparations for filing in the US are underway and we are on track to submit a New Drug Application in H1 2020.”

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