Biohaven runs a large trial testing a new compound in patients with Spinocerebellar ataxia

Biohaven Pharmaceutical Holding Company Ltd. announced end of 2016 that it has enrolled the first patient in its potentially pivotal Phase 2b/3 clinical trial assessing the efficacy and safety of its drug candidate BHV-4157 in patients with hereditary spinocerebellar ataxia (SCA). You can read the full press release here.

Posted: 20/02/2017

Interesting publication on the meanings of physiotherapy and exercise for people living with progressive cerebellar ataxia

A research team based in the Department of Clinical Sciences, College of Health and Life Sciences at Brunel University London has recently published an interesting research article aiming to understand the meanings of exercise and physiotherapy for people living with a progressive
cerebellar ataxia. You can access to the full publication here

Posted: 08/02/2017

Promising announcement about novel technology for the treatment of FA

Encouraging results of phase-2 trial in SCA3

Bioblast Pharma Ltd., a clinical-stage, orphan disease-focused biotechnology company, announced results of a six-month open label Phase 2a study that also included an additional six-month follow-up period investigating trehalose in patients with Spinocerebellar Ataxia Type 3 (SCA3). 

The key findings of the study included: (1) Weekly trehalose infusions of both doses were generally safe and well tolerated; (2) Patients remained stable over the 6-month period with no change on the Scale for Assessment and Rating of Ataxia (SARA) score; (3) Five patients received treatment for as long as 12 months and continued to remain stable on the SARA scale. You can read the full press release here

Posted: 20/01/2017 

Registration open for the International Ataxia Research Conference 2017!

Ataxia UK together with FARA and GoFAR are organising the International Ataxia Research conference in Pisa, Italy. This event is key for researchers, clinicians and representatives from pharma/biotech companies who have interest in research on ataxia. The first conference in Windsor (UK) was the largest ataxia conference ever organised and was a great success. The second International ataxia research conference will be held on 27-30 September 2017. Registration is now open; you can find more information, register and submit abstract by visiting this website.

Posted: 19/01/2017