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NPUK relies entirely on voluntary donations and fundraising to enable us to continue our work in supporting the children, adults, and families that are affected by Niemann-Pick disease. We’ve only been able to achieve what we have thanks to the voluntary donations and fundraising efforts of people like you. However, we know we can achieve much more so please continue to support us in any way you can – your kindness will help change lives.

Your donations, whether big or small, make a difference:

How you can donate:

  • If you live in the UK you can donate to NPUK easily from your mobile phone – simply text: NPUK02 followed by the amount you’d like to donate to 70070. For example e.g. “NPUK02 £5” to 70070 (UK ONLY). These donations are handled by our Just Giving account, and go directly into our fundraising account.
  • You can also donate to our Virgin Money Giving and Just Giving accounts online.

Thank you from everyone here are NPUK!

 

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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