NAF has launched a petition on Change.org calling on the FDA to prioritize treatment options for rare diseases with urgent unmet needs, including Spinocerebellar Ataxia (SCA).
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Most things about you are written in your genes: from the colour of your eyes, whether you are lactose intolerant, to if you have a higher chance of getting certain diseases. You may have heard of the popular ancestry DNA testing service that tells if you are part Chinese, part Read More…
Written by Dr. Hannah K Shorrock Edited by Dr. Hayley McLoughlin A C. elegans model of SCA3 helps to identify the serotonin receptor agonist befiradol as a potential therapeutic for SCA3 To move toward treatments for ataxia patients, it is important to understand what cellular and molecular pathways are dysfunctional Read More…
Written by Carrie Sheeler Edited by Dr. Hayley McLoughlin Researchers use Magnetic Resonance Imaging (MRI) to determine if brain volume can be a biomarker for SCA3 There are two goals of preclinical research. First, to understand the cause of a disease. Second, to develop treatments to stop or slow its Read More…
Escrito por Dr. Maria do Carmo Costa, editado por Dr. Hayley McLoughlin. Inicialmente publicado em 24 de abril de 2020. Traduzido para o português por Priscila P. Sena. Em um trabalho de colaboração utilizando modelo animal, pesquisadores de Portugal e do Reino Unido descobrem um subproduto do óleo de canola Read More…
Written by Dr Hannah K Shorrock Edited by Dr. Maria do Carmo Costa Neurofilament light chain predicts cerebellar atrophy across multiple types of spinocerebellar ataxia A team led by Alexandra Durr at the Paris Brain Institute identified that the levels of neurofilament light chain (NfL) protein are higher in SCA1, Read More…
Escrito por la Dra. Ambika Tewari Editado por la Dra. Hayley McLoughlin. Publicado inicialmente en el 6 de agosto de 2021. Traducción al español fueron hechas por FEDAES. La expresión lentiviral de un ARNhc contra ataxina-3 fue bien tolerada y no produjo efectos adversos medibles en ratones de tipo salvaje. La Read More…
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…
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…
Location: University of Minnesota, MN, USA Year Research Group Founded: 2008 What disease areas do you research? Ataxia (SCA1, SCA2, SCA3, SCA6, Friedreich Ataxia) Multiple System Atrophy – Cerebellar Ataxia Huntington’s Disease Amyotrophic Lateral Sclerosis Multiple Sclerosis Alzheimer’s disease Parkinson’s disease Traumatic Brain Injury Diabetes What models and techniques do you 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…
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