News

January 5, 2021January 5, 2021

‘Dancing at the Vatican’ Spotlights Families’ Struggles, Joy at Meeting Pope

News

The Huntington’s Disease Association of Ireland (HDAI) is spreading the word about a new documentary that tells the improbable story of Huntington’s disease (HD) families and their cross-continental journey to ... Read more

December 22, 2020December 22, 2020

Natural History Study of Huntington’s Fully Enrolled, Triplet Says

News

Triplet Therapeutics announced it has completed patient enrollment in SHIELD HD, a natural history study documenting the course of Huntington’s in the absence of treatment and associated disease biomarkers. With ... Read more

December 15, 2020December 15, 2020

Single CDNF Protein Injection Shown to Protect Nerve Cells, Improve Motor Skills in Rats

News

A single injection of the protein CDNF — cerebral dopamine neurotrophic factor — into the brains of rats with induced Huntington’s disease protected affected nerve cells from degeneration and improved ... Read more

December 8, 2020December 8, 2020

1st Patient Dosed in Phase 2a Trial of Annexon’s Antibody Therapy for Huntington’s

News

The first participant was dosed in a Phase 2a clinical trial testing Annexon’s investigational therapy ANX005 as a treatment for Huntington’s disease, the company announced. The open-label study ((NCT04514367) is currently ... Read more

December 1, 2020December 1, 2020

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

News, syndicated

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders ... Read more

December 1, 2020December 1, 2020

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

News

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 ... Read more

November 26, 2020November 26, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

News, syndicated

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to ... Read more

November 24, 2020November 24, 2020

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

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 ... Read more

November 17, 2020November 17, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

News, syndicated

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about ... Read more

November 17, 2020November 17, 2020

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

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 ... Read more