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...

CureDRPLA is funding this study to better understand the symptoms of DRPLA and how they change over time. It also aims to identify measurable indicators that could predict progression and responsiveness to treatments in future clinical trials. This study collects...

Since DRPLA is more prevalent in Japan, CureDRPLA has made it a priority to build connections with the scientific and clinical community there. Junko Shiozawa, Advisory Board Member, has played a key role in building a community of Japanese individuals affected by...

In this video we interviewed Aliza Ben-Varon, a former Graduate Student working with Dr Jeff Carroll at the University of Washington. This research group has done a fantastic job in characterizing a mouse model that mimics the human form of DRPLA. Using genetic...

Nissan Chemical Corporation and Sanwa Kagaku Kenkyusho Co., Ltd., two Japanese companies, announced on April 4 their agreement to co-develop SK-2407/SN-001, which is a pre-clinical antisense oligonucleotide (ASO) candidate identified through their collaboration for...