The free, interactive tool was developed by MRC/CSO Social and Public Health Sciences Unit, based at the University of Glasgow. It asks the user to answer a series of questions which aim to encourage critical thinking about the type and quality of the research, such as funding sources, peer review and ethics, and guide the user towards forming an evidence-based opinion about the research. To read more about it, Chris Peters, Scientific Affairs Manager at Sense about Science, has written a blog on this new online tool.
Pfizer Inc. has acquired Bamboo Therapeutics, focusing on developing gene therapies for the treatment of patients with rare diseases
This week, Pfizer Inc. announced that it has acquired Bamboo Therapeutics, a privately held biotechnology company based in Chapel Hill, NC, that is focused on developing gene therapies for the potential treatment of patients with certain rare diseases. This further highlights Pfizer Inc.’s expanded focus on exploring gene therapy for rare diseases and their strengthened research in developing potential therapies for FA. Their on-going early discovery programs in FA are complemented by the inclusion of Bamboo Therapeutics’ experimental gene therapy asset for the potential treatment of FA. You can find the press release here.
The proceedings of the International Ataxia Research Conference are published
Ataxia UK together with three others ataxia organisations (FARA, Ataxia Ireland and GoFar) organised last year the International Ataxia Research conference in Windsor, UK. This event was the largest ataxia conference ever organised and was a great success. This led to many new connections between researchers focusing on ataxia from around the world, and continues to produce significant collaborative work with the publication of the proceedings in Journal of Rare Disorders.
Ataxia UK is working in partnership with Findacure on a new way of funding trials
Ataxia UK has started a partnership with Findacure, a charity focusing on rare diseases who has developed a social impact bond (SIB) model to fund trials using repurposed drugs. Following the success of the pilot trial in FA using Nicotinamide, Ataxia UK has been working on an application for a larger trial to be funded using the Findacure SIB. We are delighted that following evaluation by Findacure’s Scientific Committee they decided to choose Ataxia UK’s project as one of three to test their model.
Updated Medical Guidlines Released
We are delighted that we are able to officially release the updated medical guidelines – for Management of the ataxias: Towards Best Clinical Practice. These guidelines were produced to help healthcare professionals in the diagnosis and management of people with ataxia. These guidelines are the result of extensive consultation with ataxia specialist neurologists and other healthcare professionals, for whose support we are extremely grateful for. They include comprehensive sections on diagnosis, medical interventions for symptom relief and therapies.