13.09.19
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)...
Read more13.09.19
We are pleased to share the programme for the upcoming 26th Annual Family Conference & 10th Interactive Workshop on Niemann-Pick Diseases. We look forward to seeing our community all together once again, if you have any issues or queries regarding this event please do not hesitate to get in touch with us by either email at: info@npuk.org or phone on: 0191 415 06 93.
Read more12.09.19
CTD is also a proud sponsor of the NPUK Annual Family Conference & Interactive Workshop on Niemann-Pick Diseases, for the 4th year running...
Read more12.09.19
Evidera, a research group working on behalf of Sanofi Genzyme, is conducting a research study to understand the symptoms and impact experienced by carers of those affected by ASMD Niemann-Pick disease types B, or A/B...
Read more30.08.19
Copenhagen, Denmark, August 28, 2019 – Orphazyme A/S (ticker: ORPHA.CO), a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announces its interim financial results for the period January 1 – June 30, 2019 and corporate highlights...
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