Huntington’s Disease News

December 1, 2020December 1, 2020

Late-onset Huntington’s Has Fewer Motor Defects, Similar Cognitive Progression, Study Shows

Patients with early-onset Huntington’s disease (HD) experience more muscle and eye movement abnormalities, and more rapid progression of motor symptoms, than those with late-onset disease, according to a recent study. ... Read more

November 24, 2020November 24, 2020

Phase 1 Trial Will Test Oral PTC518 Candidate Therapy; First Results Due Early 2021

Huntington's Disease News

A Phase 1 trial will evaluate PTC Therapeutic’s PTC518, its investigational therapy for Huntington’s disease, in healthy volunteers, the company announced. The trial’s first results are expected in the first half ... Read more

November 17, 2020November 17, 2020

PDYN-derived Peptides Identified as Possible, Much-needed HD Biomarkers

Huntington's Disease News

Levels of proteins derived from prodynorphin (PDYN) — ultimately used during cell-to-cell signaling in the brain — are decreased in the fluid surrounding the brains of people with Huntington’s disease ... Read more

November 10, 2020November 10, 2020

First Patients Enrolled in PROOF-HD Phase 3 Trial Testing Pridopidine

Huntington's Disease News

The first U.S. patients have been enrolled in PROOF-HD, a Phase 3 clinical trial investigating pridopidine as a treatment for Huntington’s disease, according to developers Prilenia Therapeutics. PROOF-HD (NCT04556656) will ... Read more

November 3, 2020November 3, 2020

1st Wexler Prize Given Scientist Studying Astrocytes Role in Huntington’s

Huntington's Disease News

The Hereditary Disease Foundation‘s (HDF) has announced the first winner of its Nancy S. Wexler Young Investigator Prize, supporting excellence and creative thinking in Huntington’s disease research. The inaugural award went to ... Read more

October 28, 2020November 30, 2020

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

Huntington's Disease News

A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in ... Read more

October 27, 2020October 27, 2020

Branaplam Receives Orphan Drug Designation for Huntington’s; Trial Planned for 2021

Huntington's Disease News

The U.S. Food and Drug Administration (FDA) has given orphan drug designation to the oral therapy branaplam (LMI070) as a potential treatment for Huntington’s disease, the therapy’s developer, Novartis, announced. The ... Read more

October 22, 2020November 25, 2020

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

Huntington's Disease 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 ... Read more

October 20, 2020October 20, 2020

FDA Puts Voyager’s Gene Therapy Candidate VY-HTT01 on Clinical Hold

Huntington's Disease News

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 ... Read more

October 13, 2020October 13, 2020

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

Huntington's Disease 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 safety, ... Read more

October 6, 2020November 6, 2020

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

Huntington's Disease News

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, ... Read more

October 6, 2020October 6, 2020

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

Huntington's Disease News

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 ... Read more

September 29, 2020September 29, 2020

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

Huntington's Disease News

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 ... Read more

September 22, 2020September 22, 2020

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

Huntington's Disease News

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 ... Read more

September 15, 2020September 15, 2020

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

Huntington's Disease News

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. ... Read more