Latest Research News - January and February 2017 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 progressivecerebellar ataxia. You can access to the full publication here. Posted: 08/02/2017 Promising announcement about novel technology for the treatment of FA Chondrial Therapeutics, an emerging biotechnology company focused on the treatment of rare mitochondrial diseases, announced today that it has secured up to $22.6 million in Series A financing. Concurrently, Chondrial announced the licensing of a novel investigational technology, CTI-1601, for the treatment of Friedreich's Ataxia from Indiana University Research and Technology Corp. (IURTC) and Wake Forest University Health Sciences. CTI-1601 was invented by R. Mark Payne, MD, professor of Pediatric Cardiology at Indiana University School of Medicine and director of the Translational Research Training Program of Indiana Clinical Translational Sciences Institute. You can read the full press release here. Posted: 08/02/2017 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
Chondrial Therapeutics, an emerging biotechnology company focused on the treatment of rare mitochondrial diseases, announced today that it has secured up to $22.6 million in Series A financing. Concurrently, Chondrial announced the licensing of a novel investigational technology, CTI-1601, for the treatment of Friedreich's Ataxia from Indiana University Research and Technology Corp. (IURTC) and Wake Forest University Health Sciences. CTI-1601 was invented by R. Mark Payne, MD, professor of Pediatric Cardiology at Indiana University School of Medicine and director of the Translational Research Training Program of Indiana Clinical Translational Sciences Institute. You can read the full press release here. Posted: 08/02/2017