Huntington’s Disease News

September 16, 2021September 16, 2021

New Institute Aims to Leave No Rare Disease Patient Behind

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest ... Read more

September 14, 2021September 14, 2021

Abnormal DNA Repair Mechanism May Be Therapeutic Target

Huntington's Disease News

The interaction between FAN1 and MLH1 — two DNA repair proteins known to be genetic modifiers of Huntington’s disease — protects against further expansion of disease-causing CAG repeats, according to ... Read more

September 8, 2021September 8, 2021

Group Focuses on Rare Disease Clinical Trial Participation

Huntington's Disease News

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running ... Read more

September 7, 2021September 7, 2021

AMT-130 Gene Therapy Appears Safe; More Data Forthcoming

Huntington's Disease News

No significant safety concerns were identified in the first eight Huntington’s disease patients treated with uniQure’s potential gene therapy AMT-130 in a U.S. Phase 1/2 clinical trial. Six of these patients ... Read more

September 3, 2021September 3, 2021

Patients, Caregivers Asked to Help Adira in Choosing Grant Awards

Huntington's Disease News

The Adira Foundation is inviting people with neurodegenerative diseases and their caregivers to join a grant proposal review committee. A nonprofit foundation, its mission is to unite people affected by some of ... Read more

August 31, 2021August 31, 2021

‘Running for Rare’: NORD a Charity Partner of 2021 NYC Marathon

Huntington's Disease News

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both ... Read more

August 31, 2021August 31, 2021

Presymptomatic Disease Changes One’s View of Time, Patients Say

Huntington's Disease News

Huntington’s disease patients who have not yet developed symptoms have a different relationship with time than otherwise-healthy individuals, according to a new study based on patient interviews. The findings highlight ... Read more

August 24, 2021August 24, 2021

‘Not Alone in the Dark’: New Guide for, by Caregivers of Children With Rare Diseases

Huntington's Disease News

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The ... Read more

August 24, 2021August 24, 2021

Glia Brain Cells May Open Door to Treatment Strategy

Huntington's Disease News

Brain cells called glia work to protect the brain from damage in Huntington’s disease by reducing the expression of genes involved in maintaining connections between nerve cells, a new study ... Read more

August 17, 2021August 17, 2021

Register Now for Global Genes’ RARE Patient Advocacy Summit

Huntington's Disease News

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending ... Read more

August 17, 2021August 17, 2021

Hearing on Huntington’s Appreciated, But Parity Act Is Priority

Huntington's Disease News

While the Huntington’s Disease Society of America (HDSA) is pleased a U.S. House of Representatives subcommittee recently held a key hearing that featured Huntington’s disease, it continues to call for ... Read more

August 10, 2021August 10, 2021

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

Huntington's Disease News

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for ... Read more

August 10, 2021August 10, 2021

VO659 Earns Orphan Drug Designation

Huntington's Disease News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Vico Therapeutics’ VO659, an investigational antisense oligonucleotide gene-silencing therapy for Huntington’s disease. “We are delighted that FDA ... Read more

August 3, 2021August 3, 2021

Advocates Lobby US Congress During Virtual Rare Disease Week

Huntington's Disease News

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the ... Read more

August 3, 2021August 3, 2021

Diagnosis Tends to Come Earlier for Those with More Education

Huntington's Disease News

People with Huntington’s disease who have had more formal education tend to be diagnosed earlier and have milder symptoms than those with less formal education, a new study indicates. The ... Read more