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Care & Support

Our main focus, above all else, is to provide unique care and support services to families and friends affected by Niemann-Pick disease (ASMD or NPC). We originally started out as a parent support group made up of ten families affected by Niemann-Pick diseases and 30 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 Clinical Nurse Specialist Laura Bell, Families Officer Louise Metcalfe, and Counselling & Wellbeing Officer Steve Neal, we work to ensure that our community feel well supported.

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Latest news


Orphazyme provides regulatory update following Type A meeting with FDA on arimoclomol in Niemann-Pick disease type C

Progress made in understanding potential resolution of topics outlined in Complete Response Letter, including need for additional data to support NDA. FDA recommends the company provides supplemental information and analyses, and the FDA offers to have further interactions to identify a path to resubmission for arimoclomol in NPC...

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