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Here at NPUK you will regularly find us working with other like-minded organisations, such as those that support other connected rare diseases and other charities who share our main concerns and hopes. We have a track-record of successful collaborations with organisations that operate in line with our focus and commitment to improving the lives of those affected with Niemann-Pick disease and other rare diseases.

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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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