News

October 13, 2020October 13, 2020

Safety Board Recommends AMT-130 Trial Continue Enrolling, Treating Patients

News

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 ... Read more

October 6, 2020October 6, 2020

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

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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 ... Read more

October 6, 2020October 6, 2020

Top-line Data Show Potential Cognitive Benefit of Pepinemab in Early Huntington’s Patients

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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. ... Read more

September 29, 2020September 29, 2020

Phase 3 Trial of Oral Pridopidine in Early Stage Patients Opening Soon

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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 ... Read more

September 22, 2020September 22, 2020

New book, ‘Harry Needs a Hug,’ Helps Children Who Have Huntington’s in Their Family

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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 ... Read more

September 15, 2020September 15, 2020

Huntington’s in Youth Closely Tied to Problems With School and Social Life

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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 ... Read more

September 8, 2020September 8, 2020

TBK1 Enzyme Seen to Clear Mutant Huntingtin Protein in Disease Models

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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 ... Read more

September 1, 2020September 1, 2020

Huntington’s Alters Brain Development Before Birth, Study Shows

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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 ... Read more

August 31, 2020September 1, 2020

Same But Different Photo Contest Celebrates People With Rare Diseases

News, syndicated

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme ... Read more

August 25, 2020September 1, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

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Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient ... Read more

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