News

November 10, 2020November 10, 2020

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

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) ... Read more

November 6, 2020November 6, 2020

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

News, syndicated

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit ... Read more

November 3, 2020November 3, 2020

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

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 ... Read more

October 28, 2020October 28, 2020

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

News, syndicated

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 ... Read more

October 27, 2020October 27, 2020

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

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. ... Read more

October 22, 2020October 21, 2020

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

News, syndicated

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 ... Read more

October 20, 2020October 20, 2020

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

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 ... Read more

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

News, syndicated

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

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. ... Read more