IntraBio announces plans for a multi-national randomised clinical trial to evaluate IB1000 as a therapeutic intervention for patients with NP-C...Read more
Take on the challenge of a lifetime from 10,000 ft! Enjoy the exhilarating and unforgettable feeling of a skydive - flying through the clouds from over 10, 000 ft at up to 120 mph! Niemann-Pick UK are looking for thrill-seeking fundraisers to take on this once in a lifetime challenge and if you raise enough sponsorship, you can jump for free! You will be strapped to your qualified British Parachute Association tandem instructor who does all the hard work for you, leaving you to enjoy the invigorating feeling of freefalling before gliding to the ground!Read more
CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease, today announced that it will host a webinar to provide details of the Company’s recently initiated Phase I clinical trial in the U.S. and Phase I/II clinical trial in the EU, both evaluating CTD’s proprietary formulation of hydroxypropyl beta cyclodextrin, Trappsol® Cyclo™, for the treatment of Niemann-Pick Disease Type C (NPC). NPC is a rare and fatal genetic disease that damages the brain, lung, liver, spleen, and other organs.Read more
On Friday 23rd June, as part of the annual MRC Festival of Medical Research, the MRC Harwell Institute will host a Patient Open Day. Patients, their families and representatives who are interested in finding out more about primary scientific research, the relationship between genes and disease, and how mice are used in medical research are invited to attend.Read more
If you or your family member(s) receive care and services linked to an ultra-rare disease, such as Niemann-Pick, we need your help.
In order to bring about change and improvement in the care and services you receive, Niemann-Pick UK has been asked to provide feedback to NHS England. By participating in a short, but highly important survey, you could help to influence this process and enhance future care for those affected by Niemann-Pick and other ultra-rare diseases.Read more