News

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

The U.S. Food and Drug Administration (FDA) is putting on hold Voyager Therapeutics’ request to test VY-HTT01, its investigational gene therapy for Huntington’s disease, in a clinical trial. The clinical hold will be kept in place until certain issues with VY-HTT01’s chemistry, manufacturing, and controls are…

No significant safety concerns associated with AMT-130, uniQure’s experimental gene therapy for Huntington’s disease, were identified in the first two patients participating in a Phase 1/2 trial assessing the treatment’s safety, tolerability, and efficacy. These were the main conclusions that came out of the first meeting of an…

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…

Vaccinex’s investigational antibody pepinemab showed good tolerability in a Phase 2 clinical trial of people with late prodromal (before clinical diagnosis) and early Huntington’s disease (HD), top-line data show. The results also hint the treatment may improve cognition; however, those efficacy results did not reach statistical significance.

Prilenia Therapeutics, in partnership with the Huntington Study Group (HSG), has launched the Phase 3 PROOF-HD clinical trial to evaluate the company’s lead experimental oral therapy, pridopidine, in people with early stage Huntington’s disease. Pridopidine “is a first-in-class [therapy] candidate, with promising previous clinical results and…

A new book is available to help children cope when a family member has Huntington’s disease. Called “Harry needs a hug: A story for children learning to deal with Huntington’s Disease,” the  32-page book was written by Emma Terranova, founder of Campaign For My Brain, a United…

Behavioral changes due to early onset Huntington’s disease and problems with school, and social and family life are common,  particularly among pediatric patients, according to a case series study in Germany. Socio-medical problems in these children were associated both with their own disease, and with the additional burden of  parents…

An enzyme called TANK‐binding kinase 1 (TBK1) can lower levels of the mutant huntingtin protein — which underlies the development of Huntington’s disease — and prevent its aggregation in cellular and animal models of the disease, a study reports. Data showed that TBK1 is able to do this by…

Huntington’s disease alters nerve cell development in the cortex — a brain region largely affected in people with the condition — before birth, a study has found. “Huntington’s definitely has a neurodevelopmental component in addition to a neurodegenerative disease,” Sandrine Humbert, PhD, the study’s senior author at the…