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Riluzole, often sold under the trade name Rilutek, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). ALS is a fatal neurodegenerative disease that mainly affects neurons controlling muscle movements. The drug was approved by the FDA (1995), Health Canada (1997), and the European Commission (1996). It Read More…
Principal Investigator: Dr. Marija Cvetanovic Location: University of Minnesota, Minneapolis, USA Year Founded: 2012 What disease areas do you research? SCA1 What models and techniques do you use? Mouse models Human stem cells Microscopy RNA Sequencing Research Focus What is your research about? The human brain is made up of many different Read More…
Written by Dr. By Marija Cvetanovic Edited by Dr. Larissa Nitschke Suart et al. show that Ataxin-1 interacts with an important DNA repair protein Ataxia telangiectasia mutated (ATM), and that reduction of ATM improves motor phenotype in the fruit fly model of SCA1, indicating DNA repair as an important modifier Read More…
Written by Sophia Leung Edited by Dr. Marija Cvetanovic While the mutant proteins in SCA19/22 lose part of their innate functions and properties, they also disrupt the key functions of the normal healthy protein. The underlying mechanism of the hereditary property of SCA19/22 is elusive. In this study, the researchers Read More…
Public transit may not be the first thing that comes to mind when we think about the brain, but it’s a great way to understand how all the parts of the central nervous system work together. Nutrients, hormones, and other important molecules (the passengers) need to get on and off Read More…
Written by Dr. Ambika Tewari Edited by Dr. Hayley McLoughlin Lentiviral expression of an shRNA against ataxin-3 was well-tolerated and produced no measurable adverse effects in wild-type mice. Evaluating the safety profile is a necessary and crucial step in qualifying a therapy for use in patients. Gene therapy is an Read More…
Principal Investigator: Dr. Sheng-Han Kuo Location: Columbia University, New York, NY, United States Year Founded: 2012 What disease areas do you research? SCA1 SCA2 SCA3 SCA6 Tremor Essential Tremor What models and techniques do you use? Mouse models Post-mortem patient tissue Optogenetics Human physiology EEG (electroencephalogram) Neuromodulation Research Focus What is your Read More…
Spinocerebellar ataxias (SCAs) are well known for worsening motor coordination symptoms caused by the degeneration of the cerebellum. Yet, increasing reports indicate that broader changes are occurring in the brains of some SCA patients. This includes changes in the hippocampus, a brain region critical for learning and memory. One way Read More…
When you think of a balance beam, you might think of gymnastics. For humans, a balance beam is a surface where we perform jumps, flips, and other athletic feats. Whether it’s a child taking their first class, or an Olympic athlete going for gold, the balance beam requires both balance Read More…
Principal Investigator: Dr. Maurizio Petrarca Location: Bambino Gesù Children Hospital, Rome, Italy Year Founded: 2000 What disease areas do you research? Friedreich’s ataxia Early-onset ataxia Non-Progressive Cerebellar Ataxia Hereditary Spastic Paraplegia Duchenne Muscular Dystrophy Charcot-Marie-Tooth disease Cerebral Palsy Adrenoleukodystrophy What models and techniques do you use? Wearable Technologies Movement analysis Read More…
