CureDRPLA was founded in November 2019 and in light of our forthcoming founding anniversary, we have prepared an impact report to summarize our work and milestones. In this report you will find a summary of our achievements to date and our goals for the next 5 years....
Attended Rare Disease Symposium
Last week, Dr Silvia Prades attended a conference to find out more about the latest work on rare diseases and how the medicines regulatory framework is expected to change in the coming years to facilitate research and approval of treatments for rare conditions like...
Paul Compton shares insights on DRPLA n-of-1 trial in n-Lorem’s podcast
CureDRPLA co-founders, Paul and Andrea Compton, have a son with DRPLA and their personal experiences with this devastating condition are the driving force behind CureDRPLA. Earlier this year, CureDRPLA announced that n-Lorem had an experimental treatment for DRPLA and...
Nissan Chemical and Sanwa Kagaku announce plans to develop drug for DRPLA
Nissan Chemical Corporation and Sanwa Kagaku Kenkyusho Co., Ltd., two Japanese companies, have announced they recently co-created an antisense oligonucleotide (ASO) for DRPLA. ASOs have emerged as a promising strategy for various neurodegenerative conditions,...
Group of volunteers organize booth at neurology conference in Tokyo
Junko Shiozawa, Advisory Board Member to CureDRPLA, has created a community of Japanese families affected by DRPLA. They offer peer support to each other and host online meetings. Since DRPLA is more common in Japan, it was important for us to create a Japanese...