Lindsey Shapiro, PhD, science writer —

PTC Therapeutics’ Huntington’s disease treatment candidate PTC518 is well-tolerated and lowers blood levels of the huntingtin protein (HTT) in people with the neurodegenerative disorder. That’s according to interim three-month data from the Phase 2a PIVOT-HD trial (NCT05358717), which found that use of the investigational oral therapy did not…

Note: This story was updated May 16, 2023, to correct the duration of ANX005 infusions. Only the first loading dose of the therapy is given for 21 hours, with subsequent doses having a duration of four to five hours. The experimental therapy ANX005 safely leads to stabilization or improvements in…

The last patient has completed the final visit in the Phase 3 PROOF-HD trial testing oral pridopidine for Huntington’s disease. That means that the study’s main placebo-controlled part is done, according to Prilenia Therapeutics, pridopidine’s developer, which sponsored the nearly 18-month-long trial. Now, the first data are…

Enrollment is underway in a Phase 2 clinical trial testing Roche’s therapeutic candidate tominersen in people with early manifest Huntington’s disease and those in the prodromal phase of the disease, when hallmark disease symptoms are not yet present. The study, dubbed GENERATION HD2 (NCT05686551), aims…

Long-term treatment with Austedo (deutetrabenazine) safely led to sustained reductions in chorea for people with Huntington’s disease, including those who had switched from treatment with Xenazine (tetrabenazine). That’s according to about three years of follow-up data in the Phase 3 ARC-HD trial (NCT01897896), a study jointly conducted…

A data safety monitoring committee has recommended that enrollment of the highest dosing group resume in the European Phase 1/2 trial of AMT-130 in people with Huntington’s disease, but with additional safety measures in place. UniQure, the investigational treatment’s developer, announced in August that enrollment in…

A single injection of Wave Life Science‘s experimental therapy WVE-003 was found, at either of the two lowest tested doses, to lead to reductions in the mutant huntingtin (mHTT) protein in people with Huntington’s disease — while leaving the healthy version of the protein unaffected. That’s according to…

Social support for children of a parent with Huntington’s disease from someone other than a caregiver is important in helping them manage stress and adversity, but there are obstacles to obtaining that support, a study has found. People who’ve lived with a parent with Huntington’s reported that, while social…

A Phase 2 trial of pepinemab failed to significantly improve certain cognitive abilities in people with prodromal to early Huntington’s disease, not meeting a primary study goal. But data indicated that diagnosed patients in early disease stages, those with existing cognitive and functional impairments at the study’s start, did…

AMT-130, uniQure’s investigational gene therapy for Huntington’s disease, was generally well-tolerated and led to reductions in the levels of mutant huntingtin protein a year after treatment, according to an update from an ongoing Phase 1/2 trial. Moreover, levels of neurofilament light (NfL), a biomarker of nerve damage —…