Bridge the communication gap: collaborate with patients and patient advocacy groups for more efficient, quality-driven trials
Ataxia UK’s Research Officer was invited to take part in a panel of discussion at the recent Clinical Excellence Europe 2016 organised in London by the company eyeforpharma. You can find the full programme here. Dr Vallortigara had the opportunity to raise awareness about ataxia and present Ataxia UK’s mission in a brief talk before joining a Q&A session. The presentation is available here and you can listen to the discussion by following this link.
Research highlights from National Ataxia Foundation annual meeting
Ataxia UK’s CEO Sue Millman together with the CEO of Ataxia Ireland Barbara Flynn attended the last edition of NAF annual meeting. You can read a summary of the conference here and you can access the presentations by visiting this page.
Scotland has joined the 100,000 Genomes project
The 100,000 Genomes Project continues to gather pace and several new projects and partnerships have been launched in the last few months. The great news is that Scotland has joined the 100,000 Genomes Project. A £6m investment in the Scottish Genomes Partnership means rare disease patients in Scotland will soon be able to take part in the Project. You can read more about the Scottish Genomes Partnership on the Genomics England website here.
The Scripps Research Insitute has published interesting results on SCA10
In an important new study with implications for the treatment of dozens of incurable diseases, scientists from the Florida campus of The Scripps Research Institute (TSRI) have for the first time created a drug candidate that attacks and neutralizes the RNA structure. The study, which was published June 1, 2016 in Nature Communications, showed the compound significantly improved several aspects of cells taken directly from patients with spinocerebellar ataxia type 10 (SCA10), a form of spinocerebellar ataxia. To read more about this study, please visit this page.
The company Horizon Pharma has bought the right for Interferon Gamma in Europe
Horizon Pharma is running a phase-III trial in USA using Interferon gamma and completed the recruitment of 90 patients with Friedreich’s ataxia early this month. They have now announced the agreement with Boehringer Ingelheim International GmbH to buy their rights for this drug in Europe and Middle East. This is very promising for a future trial and potential use of this drug in Europe if this trial is successful – to read more please visit their newspage here.