cure drpla

CureDRPLA Global Patient Registry is available!

Click here for global patient registry

What is DRPLA?

Dentatorubral-pallidoluysian atrophy, commonly known as DRPLA, is a progressive brain disorder that affects coordination, balance, speech, and causes involuntary movements, emotional problems, and a decline in thinking ability. The average age of onset of DRPLA is 30 years, but this condition can appear anytime from infancy to adulthood. The symptoms and progression vary depending on the age of onset.

What is CureDRPLA?

CureDRPLA is a US based non-profit founded by Paul and Andrea Compton. Paul and Andrea have a son that was diagnosed with DRPLA in August 2018. The mission of CureDRPLA is to connect families, physicians and scientific investigators to further DRPLA research and work towards a treatment for DRPLA. This webpage was created to find others that have this condition and are interested in finding a cure.

CureDRPLA
Global
Patient
Registry

The CureDRPLA Global Patient Registry is a worldwide registry of DRPLA patients.

This registry gathers the demographic and clinical information on DRPLA patients from across the globe.  By collecting information on all DRPLA patients, the CureDRPLA Global Patient Registry will be a powerful resource for research and will enhance opportunities for treatment development.

Patient Registry

Patient & Caregiver

Click below to find out what CureDRPLA has done and what we are currently doing to find to a treatment for DRPLA.  You will also find online resources and support organizations for DRPLA as well as YouTube channels specifically focused on DRPLA.

Patient & Caregiver

Click below to find out what CureDRPLA has done and what we are currently doing to find a treatment for DRPLA.  You will also find online resources and support organizations for DRPLA as well as YouTube channels specifically focused on DRPLA.

Patient & Caregiver

Patient &
Caregiver

Researcher
& Industry

CureDRPLA is investing in a number of projects in hopes of rapidly developing novel therapeutics for DRPLA.  Information on funding opportunities, a list of our preclinical projects as well as other efforts can be found here.

Research & Industry

Latest

Upcoming webinars on DRPLA with the National Ataxia Foundation

We are co-hosting a couple of webinars on DRPLA with the National Ataxia Foundation (NAF) to reach as many people as possible. We encourage anyone with DRPLA, family members, caregivers and friends to join to learn more. The “All About DRPLA” webinar is taking place...

Watch the recording of the 2025 DRPLA Research Update

We recorded our annual update so you can watch it at your own pace. It includes updates on our organization, N-of-1 trials, DRPLA Natural History and Biomarkers Study, Patient Registry and other relevant news and projects.

25th September is International Ataxia Awareness Day (IAAD)

This year we are raising awareness about DRPLA. Let’s come together to share our 5-Year Progress Report, which includes:✔️ A comprehensive summary of DRPLA✔️ Highlights of research projects funded by CureDRPLA✔️ Updates on efforts to advance treatments for...

Prof Darren Monckton joins CureDRPLA as Scientific Consultant

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...

5 years of CureDRPLA

CureDRPLA was incorporated as a non-profit foundation in November 2019 by Paul and Andrea Compton. We are honoring 5 years of CureDRPLA with this impact report. In this report you will find a summary of our achievements to date and our goals for the next 5...

Join us at ICAR – November 12-15

ICAR, largest gathering of ataxia scientists and medical professionals, will take place in London, November 12-15. CureDRPLA team members will be there, along with scientists working on DRPLA who will present their latest research.

DRPLA n-of-1 clinical trial

Take a few minutes to read this announcement about an upcoming n-of-1 clinical trial for an individual with DRPLA based in the US.

Watch our 2023 DRPLA research update

Every year, we like to provide an update on our projects, offering you insight into what we are working on and what is on the horizon. Do not miss out! https://youtu.be/9R5UcB26_FU

Register for DRPLA Research Talk and Q&A, Nov 14

Event tailored for individuals with DRPLA, their families and anyone interested in DRPLA. Make sure to register in advance (it’s free!). Our Research Manager, Dr Silvia Prades, will be providing a comprehensive update on the projects funded by CureDRPLA. Followed by a...

The 2023 CureDRPLA Research Conference is taking place in Boston on October 10-11

We hosted the first-ever CureDRPLA Research Conference in May 2022, with participation from scientists, neurologists, and representatives from pharmaceutical and biotech companies. It was such a huge success that we decided to make it a yearly event and the time has...

News

Announcing DRPLA n-of-1 clinical trial

Announcing DRPLA n-of-1 clinical trial

May 28, 2024

This announcement was initially published on our website on February 12, 2024. You can find a pdf version of the announcement here. Dear DRPLA community, Since we established CureDRPLA in 2019, our mission has been to connect families, physicians and scientific...

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Social

Cure DRPLA

1 week ago

Cure DRPLA
In March we reported that Prof Jeff Carroll, our longstanding Scientific Advisor, resigned to pursue new opportunities. CureDRPLA is committed to keeping our team strong and ensuring we have the right scientific input to advance research towards treatments. With that in mind, we are pleased to announce that we have appointed Dr Joanna (Asia) Korecka-Roet as a Scientific Advisor for CureDRPLA. Dr Asia Korecka-Roet is an Assistant Professor at the Neurology Department at Mass General Brigham (Boston, US). Her research specializes in validating and de-risking genetic medicine for neurodegenerative disorders. She brings years of experience in DRPLA research, having developed advanced patient-derived cell lines for studying DRPLA in the dish. She has used these lines to screen ASOs for DRPLA, as well as to study epilepsy and test whether ASOs can rescue seizure-like behavior in cells. Asia joins Prof Darren Monckton, who is also a Scientific Advisor for CureDRPLA. Darren is a Professor of Human Genetics at the University of Glasgow (UK). His expertise is on repeat expansion disorders like DRPLA, in which some parts of the DNA are unusually long and unstable, which can worsen over time and generations. His research group is working to understand why this happens by studying human samples, lab models and computer simulations. ... See MoreSee Less
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Cure DRPLA

1 month ago

Cure DRPLA
If you missed the “Research and Treatment Development for DRPLA” webinar we co-hosted with NAF on May 21, you can now watch the recording on Youtube. This session was presented by Dr Silvia Prades who works at CureDRPLA and Ataxia UK ... See MoreSee Less

Research and Treatment Development for DRPLA

youtu.be

During this session, our expert will teach us how DRPLA is studied ...

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Cure DRPLA

2 months ago

Cure DRPLA
If you missed the "All About DRPLA" webinar we co-hosted with National Ataxia Foundation on May 8, you can now watch the recording on Youtube. This session was presented by Dr Yael Shiloh-Malawsky, Professor of Neurology at the University of North Carolina at Chapel Hill ... See MoreSee Less

All About DRPLA

youtu.be

During this webinar, experts will discuss Dentatorubral-Pallidoluysian Atrophy (DRPLA), the typical diagnostic journey for those affected, what to expect for...

Video

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Cure DRPLA

2 months ago

Cure DRPLA
Reminder! CureDRPLA and NAF are co-hosting a couple of webinars on DRPLA. The “Research and Treatment Development for DRPLA” webinar is taking place on May 21 at 10am CDT / 4pm BST. This session will be presented by Dr Silvia Prades, Research and Business Operations Manager for the DRPLA Research Programme at CureDRPLA and Ataxia UK. You can register for this session here: www.ataxia.org/event/research-and-treatment-development-for-drpla/ ... See MoreSee Less
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Cure DRPLA

2 months ago

Cure DRPLA
Speech can be affected in people with DRPLA and have a big impact on daily life. If you live in the UK, there are a couple of resources that might be of interest:Speech TherapyProfessor Anja Lowit and Ataxia UK have developed a six‑week online speech therapy course tailored for people with ataxia. For more information visit: www.ataxia.org.uk/support-services/speech-therapy/The course includes a mix of individual and group sessions delivered online. Due to high demand, there is currently a waiting list. To express interest, contact: volunteering@ataxia.org.ukVoice BankingAtaxia UK offers funding for voice banking through SpeakUnique. This service allows people with ataxia to create a personalised synthetic voice that can be used on a communication device if their speech becomes harder to understand.For more information visit: www.ataxia.org.uk/support-services/voice-banking/ ... See MoreSee Less
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CureDRPLA Global Patient Registry

The CureDRPLA Global Patient Registry aims to collect data on up to as many DRPLA patients as possible. Participants will be asked to complete a set of questionnaires after enrollment with email requests for yearly updates. Data collected include participant demographic and contact information, details about diagnosis, functional mobility status, health economics, medical history, and activities of daily living. 

Global Patient Registry