28.02.17
'With the AIDNPC study close to being fully recruited, Orphazyme proposed a change of frequency for the now-monthly teleconference calls providing updates. Once the last patient has been enrolled, the updates could be provided on a quarterly basis. The company invites the patient organisations to share their views on the proposed change in TC frequency.'
Read more30.01.17
Niemann-Pick UK and the International Niemann Pick Disease Alliance would like to congratulate Aparito on celebrating two successful years of working in partnership with patient organisations, hospitals and research institutions across the World!
Read more17.01.17
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Trappsol® Cyclo™ for the treatment of systemic Niemann-Pick Disease Type C (NPC), a rare and fatal genetic disease. Trappsol® Cyclo™ is the Company's proprietary formulation of hydroxypropyl beta cyclodextrin in development for the treatment of NPC; it has previously been designated an Orphan Drug by the FDA and the European Medicines Agency.
Read more04.01.17
Jim Green, a founding member of NPUK all the way back in 1991 - and therefore an individual who has been connected to Niemann-Pick UK for over 25 years, is stepping down from the charity's Board of Trustees. ..
Read more07.11.16
Oxford, UK – 3 November, 2016 – Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today that the first adult patient in the UK has enrolled and been dosed in a pivotal Phase 2/3 clinical trial named ASCEND for the investigational therapy olipudase alfa. Olipudase alfa is an enzyme replacement therapy being studied for the treatment of non-neurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B (NPD B). The first patient was dosed at the National Hospital for Neurology and Neurosurgery at the University College of London Hospitals (UCLH).
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