Cognitive disorders, on the other hand ... When the HD mutation was eventually identified as a CAG trinucleotide repeat expansion, HD joined a novel class of neurodegenerative disease, the ...

It is an incapacitating disorder without cure ... 2025A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington’s disease chromosomes. Cell.

Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative ...

Huntington's is an autosomal dominant genetic disorder caused by extended CAG trinucleotide repeats in the huntingtin (HTT) gene. The sequence tends to expand continually in a process known as ...

The expansion of CAG trinucleotide repeats located in the coding sequence (CDS) of various human genes is associated with the development of neurodegenerative disorders (NDs), including Huntington’s ...

Melissa Beiner, M.D., SCA Clinical Development Lead at Biohaven, commented, Our NDA filing is the culmination of over 8 years of clinical research a ...

Spinocerebellar Ataxia (SCA) is a rare, genetic, life-threatening neurodegenerative disease with no available treatment. Troriluzole demonstrated a 50-70% slowing of SCA disease progression ...

On further evaluation, including neurology and genetics consultation and workup, a clinical diagnosis of the neurodegenerative disorder Huntington’s ... specifically looking for trinucleotide repeats.

"This is a really different way of thinking about how a mutation brings about a disease, and we think that it will apply in DNA-repeat disorders beyond Huntington's disease." "The point of our ...