Huntington's Disease Gene Also Enhances Early Brain Development ... potential cause of the genetic mutations that result in rare neurodegenerative ... Drug Significantly Reduces Chorea Symptoms ...

HD is a rare, adult-onset, autosomal dominant, progressive neurodegenerative disease. George Huntington (Figure 1) was the first person to provide a comprehensive description of adult-onset HD in ...

Doctors diagnosed Carmen Lopez with spinal muscle atrophy (SMA) at birth, a rare genetic disorder that affects motor neurons ...

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Using RNA to interfere with a gene’s expression of disease-causing proteins is a validated therapeutic approach, but so far, the products in this drug class only address liver proteins.

Study uncovers how gradual CAG repeat expansion in neurons drives the onset of Huntington’s disease, offering new insights for potential therapeutic interventions. Study: Long somatic DNA-repeat ...

Less than a month after researchers unveiled that a “ticking DNA clock” is behind Huntington’s disease (HD), scientists have now identified an antisense oligonucleotide (ASO) that can slow ...

Huntington's disease (HD) is a hereditary neurodegenerative disorder caused by a mutation in the gene called huntingtin on chromosome 4. HD is inherited in an autosomal dominant fashion, so that each ...