"Completing enrollment in the pivotal Phase 2b/3 clinical trial of VTS-270 is a significant milestone for the NPC community and Vtesse, and we are fortunate to have exceeded our goal of 51 participants. We are extremely grateful to the study participants and their families, as well as the clinical site investigators and staff, for their collective commitment and significant contributions to the development of a potential treatment for NPC," said Ben Machielse, Drs., Vtesse's President and Chief Executive Officer. "We look forward to generating topline results from the study early next year."Read more
'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 more
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 more
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 more