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What is a Randomized Controlled Trial (RCT)? Before a new drug or therapy can reach patients, researchers and regulatory agencies need evidence that it is safe and effective. One of the most reliable ways to gather this evidence is through a randomized controlled trial (RCT). A key challenge in medical Read More…
Written by Jamie Hyde Edited by Marija Cvetanovic, PhD “A new matched stem cell model gives SCA1 researchers a clearer way to study disease mechanisms and test potential treatments.” Using human stem cells to model SCA1 Without the right comparison, even the best disease model can give unclear answers. Spinocerebellar Read More…
Written by Juan MatoEdited by Jacen Emerson A new drug may help steady movement and improve daily life for people with ataxia-telangiectasia. Small improvements in motor control can have massive effects for individuals living with ataxia-telangiectasia, a rare inherited neurodegenerative disease with no current FDA- approved treatment. Ataxia-telangiectasia, affecting 1 Read More…
The functional Scale for the Assessment and Rating of Ataxia (f-SARA) is a scoring system to track how much a person’s balance and movement are affected. The f-SARA is an updated version of the original scale for ataxia, also known as SARA, especially designed for Spinocerebellar Ataxia. Traditionally, doctors measure Read More…
Written by Ray Truant, PhDEdited by Celeste Suart, PhD How the gut helps clean the brain In 2013, neuroscientists used mouse models to show that during deep Random Eye Movement (REM) sleep, the structure of the brain changes, opens up like a sponge to help clear out metabolites and misfolded Read More…
Written by Anastasiya Potapenko Edited by Asmer Aliyeva Tiny tags, big impact: new research reveals chemical tags on ataxin-3 protein influences its behavior and may contribute to brain cell death in SCA3. The genetic cause of spinocerebellar ataxia type 3 (SCA3) was discovered over three decades ago. Since then, scientists Read More…
(The Hauser Lab Back row, left to right: Linus Wiora, Stefan Hauser, Jacob Helm, Hariharan Menon, Luisa Baraban, Anabel Dickemann, Egemen Turkyilmaz. Front row, left to right: Lukas Senftleben, Natalia Vingut, Benedikt Meyer, Amelie Leonhardt, Clemens Sauter, Till Stoltenow. Not pictured: Yvonne Schelling, Melanie Kraft, Ludger Schöls) Principal Investigator: Stefan Hauser, PhD (Dr. rer. nat.) Location: German Center for Neurodegenerative Diseases, Eberhard-Karls-University of Tübingen, Tübingen, Germany Year Founded: 2023 What Read More…
When scientists test a new treatment in a clinical trial, they need a reliable way to determine whether it is safe and effective. To do this, they rely on endpoints. Endpoints are clearly defined outcomes that are measured during a trial to help determine the effects of an intervention. Clinical trials usually include several types of Read More…
Levacetylleucine, sold under the brand name Aqneursa, is an FDA-approved treatment for Niemann-Pick disease type C (NPC). It is an oral medicine, approved by the FDA in September 2024, later approved in the European Union in January 2026. Its exact mechanism of action remains unclear, but it is believed to Read More…
Written by Tala Ortiz Edited by Celeste Suart, PhD Ever wonder if experiences early in life impact symptoms of a genetic disease? Researchers found that genetics isn’t the only thing to consider in SCA6 Does the environment play a role in genetic diseases? That is the question these authors aim to answer in the context Read More…
