10.02.17
On 28 February 2017, the tenth edition of Rare Disease Day will see thousands of people from all over the world come together to advocate for more research on rare diseases. Over the last few decades, funds dedicated to rare disease research have increased. But it can’t stop there.
Rare Disease Day 2017 is therefore an opportunity to call upon researchers, universities, students, companies, policy makers and clinicians to do more research and to make them aware of the importance of research for the rare disease community.
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 more30.11.16
Vtesse announced early in 2016 that the FDA granted Breakthrough Therapy designation status for VTS-270, which is currently in a pivotal Phase 2b/3 clinical trial in NPC. Both the FDA and the European Medicines Agency (EMA) previously granted Orphan Drug status to VTS-270, and the United Kingdom's Medicines and Healthcare Products Regulatory Agency (MHRA) granted it a Promising Innovative Medicine (PIM) designation.
"We are incredibly grateful to receive Rare Pediatric Disease designation status from the FDA for VTS-270, in addition to the Breakthrough Therapy designation status, Orphan Drug designations, and the MHRA PIM designation. These designations are granted to drugs with high promise that may address areas of unmet medical need or for rare diseases. These designations will aid us as we seek to develop VTS-270 as an effective treatment to slow the progress of NPC or stabilize patients who suffer from the devastating impacts of NPC," said Ben Machielse, Drs., President and Chief Executive Officer of Vtesse. "We recognize the considerable collaboration and efforts to advance the clinical development and regulatory processes in the rare disease space, and, above all, we continue to thank all of the parents who have supported the development of VTS-270."
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