In the past, we have reported that the US is further ahead with individualized genetic therapies and N-of-1 trials for ultra rare conditions such as DRPLA because established systems and frameworks enable this work. It is encouraging to see other countries, such as...
Did you miss the meeting on November 12? We recorded the presentation so you can watch it at your own pace. The video is available on our YouTube channel. This talk includes updates on our organization, N-of-1 trials, DRPLA Natural History and Biomarkers Study,...
The most comprehensive description of symptoms and findings from clinical tests, brain scans, and blood tests from people living with DRPLA has been published by Dr Shi-Rui Gan and his collaborators based in China. They enrolled 116 people living with DRPLA across...
Did you know that September 25 is International Ataxia Awareness Day? It takes place on September 25 of each year to increase public knowledge and understanding of all types of ataxia. On this International Ataxia Awareness Day (IAAD) we are encouraged by all the...
Scientists created a mouse model with the genetic change that causes DRPLA in people. These mice showed symptoms common of DRPLA, such as balance problems, seizures, tremors, and poor coordination. When the mice were treated with a type of genetic therapy called...