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.
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Thank you from everyone here are NPUK!
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.”Read more