The European Reference Networks (ERNs)

In 2005 a taskforce started working on the European Reference Networks for rare neurological diseases. The intention of the network is to facilitate improvements in access to diagnosis, treatment and provision of high-quality, accessible and cost-effective healthcare for patients who have a medical condition requiring a particular concentration of expertise. The networks will also be focal points for medical training and research, information dissemination and evaluation.

On December 15, the European Commission approved the first 23 European Reference Networks for rare diseases (ERNs), including the one covering the ataxias, which is acknowledged to be one of the best examples. You can read their press release here:

First 23 European Reference Networks for Rare Diseases - A milestone for 30 million patients in Europe

Posted: 20/12/2016 

Disappointing results of Horizon pharma's trial testing ACTIMMUNE in FA

Horizon Pharma’s Phase 3 clinical trial, STEADFAST, evaluating ACTIMMUNE for Friedreich’s ataxia (FA) did not meet its primary endpoint.  In addition, the secondary endpoints did not meet statistical significance.  Based on the disappointing trial results the company announced that the FA development program will be discontinued. See press release here for more information.  

Posted: 09/12/2016