According to the University of California San Francisco, Huntington’s disease affects one in every 10,000 to 20,000 people in ...

Huntington’s disease is an inherited neurological disorder causing movement, cognitive, and behavioral issues. Effective management includes medical treatments, therapies, nutritional support ...

LETI-101 has demonstrated therapeutically relevant reductions of mutant huntingtin (HTT) protein, exceeding 80%, while preserving essential ...

Rare diseases (RD) are more common than you might think. For every 20 people you know, one is likely affected by a rare disease. Of the 7,000 known RD conditions, 80% have a genetic origin, yet over ...

The mutation in the gene that encodes the huntingtin ... of disease progression as measured by composite Unified Huntington's Disease Rating Scale (cUHDRS) compared to an external control (p ...

At birth, people who carry a string of more than 40 CAG repeats within the first exon of the huntingtin gene are all but destined to develop Huntington’s ... repeats could stave off the disease among ...

High-throughput genetic testing identifies 25% more rare disease risks, empowering families with early detection & prevention. Learn more this World Rare Disease Day!

In practice, neither am I. Though I’ve got a rich scientific education, it would be more than a stretch to say I actually ...

Welcome to the PTC Therapeutics fourth quarter and full year 2024 earnings conference call. (Operator Instructions) Today's conference is being recorded. I would now like to turn the conference over ...