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Genetic treatment protects against DRPLA symptoms in a mouse model with DRPLA

Genetic treatment protects against DRPLA symptoms in a mouse model with DRPLA

by Silvia Prades | Jul 31, 2025 | News Articles

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...

Prof Darren Monckton joins CureDRPLA as Scientific Consultant

by Silvia Prades | Jul 30, 2025 | Announcements

Darren Monckton is a Professor of Human Genetics at the University of Glasgow and he will be advising CureDRPLA on scientific matters. Prof Monckton brings years of experience in research for conditions like Huntington’s disease and Myotonic Dystrophy. His expertise...
Recruitment update for the DRPLA Natural History and Biomarkers Study

Recruitment update for the DRPLA Natural History and Biomarkers Study

by Silvia Prades | Jul 30, 2025 | News Articles

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...
Strengthening ties with Japan’s rare disease community

Strengthening ties with Japan’s rare disease community

by Silvia Prades | Jul 30, 2025 | News Articles

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...
Characterizing DRPLA mouse model and testing potential therapeutics – an update from Dr Jeff Carroll’s group

Characterizing DRPLA mouse model and testing potential therapeutics – an update from Dr Jeff Carroll’s group

by Silvia Prades | Apr 16, 2025 | News Articles

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...
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Recent Posts

  • Access the recordings of the latest DRPLA webinars
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  • Upcoming webinars on DRPLA with the National Ataxia Foundation
  • Presenting DRPLA research projects at conferences
  • Information on genetic testing  

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