A clinical trial conducted by Prof. Alexandra Durr's team (Sorbonne University.AP-HP) at the Paris Brain Institute and the Pitié-Salpêtrière Hospital AP-HP shows that despite the hopes raised in ...
We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. The trial will evaluate VO659, an antisense ...
A team led by Specially Appointed Associate Professor Tomohiko Ishihara and Professor Osamu Onodera at Niigata University, along with Professor Yoshitaka Nagai at Kindai University, conducted a ...
We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Back to Healio Riluzole did not improve clinical or ...
Researchers at the RIKEN Center for Developmental Biology have succeeded in creating a new model system that can be used to develop drug therapies for genetic disorders like spinocerebellar ataxia ...
Spinocerebellar ataxias are a group of neurodegenerative diseases characterized by the degeneration of Purkinje cells, a major class of neurons in the cerebellum. The resulting cerebellar dysfunction ...
Spinocerebellar ataxia (SCA) has several genetically defined subtypes. A recent multicenter study investigated the clinical symptoms associated with these subtypes, to identify patterns of clinical ...
Cure Rare Disease (CRD), a clinical-stage nonprofit biotechnology company developing genetic medicines for ultra-rare and rare conditions, today announced that the U.S. Food and Drug Administration ...
Neurodegenerative disorders like Alzheimer’s and Parkinson’s disease result from the loss of specific types of neurons due to abnormal accumulation of mutant proteins. Although specific brain regions ...
Disruptive clumps of mutated protein are often blamed for clogging cells and interfering with brain function in patients with the neurodegenerative diseases known as spinocerebellar ataxias. But a new ...
Spinocerebellar ataxia type 7 (SCA7) is a neuro-degenerative disorder characterised by progressive cerebellar ataxia and macular degeneration. SCA7 is one of the least common genetically verified ...
VO659 is the only clinical-stage candidate targeting the CAG repeat expansion that causes these diseases VO659 was designed to preferentially reduce mutant HTT and spare wild-type HTT Phase 1/2a ...
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