This year we returned to Wyboston Lakes, Bedfordshire, over the weekend of 20th-22nd September 2019, to celebrate our 26th Conference! Wyboston Lakes is one of our favourite locations, with many memories made over the years, and we look forward to welcoming friends old and new, those newly diagnosed, living with Niemann-Pick or those that have experienced bereavement. This year’s Conference once again offered the opportunity to hear the latest developments regarding therapies and clinical trials for ASMD Niemann-Pick disease types A and B, and Niemann-Pick disease type C, plus related care issues, breakout sessions and workshops. Individuals and families wishing to attend should fill out the form, here.
The Interactive Workshop on Niemann-Pick Diseases, an event for professional delegates only, took place on Friday 21st September. Hosted by Professor Fran Platt, University of Oxford, the meeting will feature clinical and scientific presentations and workshops on relevant topics, including clinical management, research and potential therapies. Clinicians and health professionals wishing to attend, should fill out the form here.
If you would like further information on the Annual Family Conference & Interactive Workshop please contact NPUK directly by email at: info@npuk.org, or by phone on: 0191 415 0693.
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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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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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