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UK Specialist Centres

Specialist care centres can provide comprehensive, integrated, multidisciplinary care for patients, as well as information and support for family members. They aim to incorporate networks of all relevant medical disciplines within the core team.  They have effective links with national networks of testing laboratories and other care centres at a national and international level.

Specialist nurses play a vital role in the day-to-day running of clinics and deal with many of the familial aspects of work with patients and family members. Physical therapists, speech therapists, occupational therapists and disease counsellors should also all be involved in supportive care for patients.

Everyone affected by Niemann-Pick disease in England is entitled to receive their clinical care at a Specialist Centre: 

Adult Niemann-Pick Disease Patient Centres:

Paediatric Niemann-Pick Disease Patient Centres:

Scotland, Wales and Northern Ireland:

Arrangements are in place for patients from Scotland, Wales and Northern Ireland to be seen within the designated centres in England for diagnosis and clinical management.  Decisions on funding treatment/access remain with the patient’s local health authority:

For further information visit the NHS Specialised Service Website.

specialist

Latest news

13.09.19

CTD Holdings Announces FDA Approval of US Expanded Access Treatment Program in Niemann-Pick Disease Type C

ALACHUA, FL–(Businesswire)–13 September 2019–CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, today announced that the company will provide Trappsol® Cyclo™, its proprietary hydroxypropyl beta cyclodextrin drug, to a pediatric patient diagnosed with Niemann-Pick Disease Type C...

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13.09.19

IntraBio: Clinical Trial IB1001-201: US Recruitment

IB1001-201 is a multinational study that investigates the effects of the novel drug IB1001 (N-Acetyl-LLeucine) for treatment of Niemann-Pick Disease type C (NPC). The trial has previously been accepted by Independent Ethics Committees (IEC), and the UK Medicines and Healthcare products Regulatory Agency (EudraCT: 2018-004431-71), as well as National Regulatory Agencies in Germany, Slovakia, and Spain, and the US Food and Drug Administration (IND 134369)...

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