Groundbreaking Progress Made in Huntington’s Disease Therapy

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00:00Groundbreaking progress made in Huntington's disease therapy.

00:04The landscape of treating Huntington's disease, a cruel neurological disorder,

00:09may be on the verge of a revolutionary shift.

00:12Huntington's is a rare, inherited condition caused by a genetic mutation,

00:16leading to toxic protein production and progressive brain cell destruction.

00:21As an autosomal dominant disease,

00:23children of affected parents have a 50% chance of inheritance.

00:26Unicure's gene therapy candidate AMT130, delivered directly to the brain,

00:34aims to silence this toxic protein.

00:36In a groundbreaking Phase 1-2 study,

00:40the high-dose cohort demonstrated a statistically significant 75% slowing

00:45of disease progression compared to controls,

00:47and a 60% slowing in functional decline.

00:50Supportive biomarker data showed that cerebrospinal fluid neurofilament light protein,

00:55which rises with neurodegeneration, remain below baseline in treated patients.

01:01This represents a historic turning point,

01:03as current treatments only manage symptoms rather than modifying disease progression.

01:08For patients and families who previously faced inevitable decline in motor function,

01:13cognitive abilities, and psychiatric health,

01:15this offers unprecedented hope.

01:18HD typically manifests between ages 30 to 50,

01:21worsening over 10 to 25 years.

01:23The therapy could preserve independence significantly longer than previously possible.

01:29Unicure plans to submit for FDA approval in early 2026,

01:34with potential launch later that year.

01:36Flat

01:52Zipper

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