Huntington Disease

David Stamler, MD, Chief Executive Officer of Alterity Therapeutics, discusses the FDA's alignment on the pivotal Phase 3 program for ATH434 in multiple system atrophy and what a successful trial could mean for a disease with no approved disease-modifying therapies.

Leslie M. Thompson, PhD; Ravi Rajmohan, MD, and Jefferson W. Chen, MD, from UCI Health discuss the rationale, delivery, and early steps of REGEN4HD, the world's first neural stem cell phase 1b/2a clinical trial for Huntington disease.

Findings from a 25-year analysis showed that tiapride was associated with significantly fewer adverse effect–related discontinuations than tetrabenazine in patients with Huntington disease chorea.

Twelve-month phase 1/2 data show SKY-0515-treated patients trending favorably versus natural history expectations across motor, functional, and cognitive endpoints, while 65% of patients and 50% of clinicians reported disease improvement.

New preclinical findings suggest that SOM3355's combined VMAT1 inhibition, VMAT2 modulation, and β1-adrenergic antagonism may offer a differentiated approach to managing motor and neuropsychiatric symptoms in Huntington disease.

The FDA indicated that 3-year phase 1/2 data may support a BLA submission for AMT-130, an investigational gene therapy that previously demonstrated significant slowing of Huntington disease progression in treated patients.

The professor of psychiatry and neurology reviewed approaches to treating chorea, cognitive impairment, apathy, irritability, and aggression in Huntington disease, while highlighting the promise of huntingtin-lowering therapies.

Real-world survey data suggest deutetrabenazine treatment may be associated with improvements in patient- and caregiver-reported quality-of-life outcomes among individuals with Huntington disease chorea.

12-montn interim findings from an ongoing phase 1/2 study showed up to 69% reductions in mutant huntingtin protein and favorable cUHDRS trends with the investigational oral RNA-splicing modifier SKY-0515.

In honor of Huntington Disease Awareness Month, held annually in May, the director of the Huntington's Disease Center at Northwestern Medicine discussed the design of the INVEST-HD trial.

Interim results from the PIVOT-HD extension study suggested dose-dependent effects of votoplam on measures of disease progression in stage 2 Huntington disease compared with a natural history cohort.

Early-phase 1b trial data showed that ANX005 is generally safe and engages its complement target in Huntington disease.

Neurocrine Biosciences launches a phase 2 trial for NBI-1065890, targeting tardive dyskinesia with promising new treatment options.

Head-to-head PET imaging data indicated that valbenazine achieves significantly higher VMAT2 target occupancy than deutetrabenazine XR at therapeutic doses, with both treatments remaining well tolerated.

uniQure navigates FDA's unexpected shift on AMT-130, aiming for accelerated approval for Huntington's disease despite recent regulatory challenges.

Real-world data from Teva Pharmaceuticals’ IMPACT-TD Registry showed that deutetrabenazine and its extended-release formulation led to significant reductions of involuntary movements and improved quality of life for adults with tardive dyskinesia.

A new study presented at the National Society of Genetic Counselors Annual Conference highlights strong interest among individuals at risk for Huntington disease in undergoing modifier gene testing, underscoring the growing role of genetic insights in personal and clinical decision-making.

UniQure's AMT-130 shows promise in slowing Huntington disease progression, paving the way for a potential breakthrough treatment by 2026.

SKY-0515 shows promising results in reducing mutant huntingtin protein in Huntington disease patients, paving the way for transformative treatment options.

Skyhawk Therapeutics initiates FALCON-HD trial for SKY-0515, a promising treatment for Huntington disease, aiming to modify disease progression.

The phase 2 trial tests Roche’s investigational antisense oligonucleotide therapy tominersen in patients with early stages of Huntington disease, a disease with no FDA-approved therapies available.

Leveraging the Enroll-HD dataset, recent data revealed consistent links between antidopaminergic medication use and worsened cognitive and motor outcomes in Huntington disease.

New 24-month data further showed dose-dependent lowering of neurofilament light and trends on major end points such as cUHDRS, Total Function Capacity, and Symbol Digit Modalities Test.

Roche has updated its phase 2 GENERATION HD2 trial to continue testing only the higher 100 mg dose of tominersen, following interim data favoring its potential clinical benefit in Huntington disease.

A recent study reported that silmitasertib, an investigational drug currently in development for cancer, may help reduce motor deficits and neuropathology in a Huntington disease.