News

SAGE-718 on FDA Fast Track as Potential Huntington’s Therapy

The U.S. Food and Drug Administration (FDA) has granted fast track designation to SAGE-718 as a potential therapy for Huntington’s disease. The FDA gives this designation to experimental medications with the potential to improve medical care for serious health conditions and fill unmet needs. The designation gives the therapy’s…

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 therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

Abnormal DNA Repair Mechanism May Be Therapeutic Target

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 a study using human and mouse models of the disease. Specifically, this interaction prevents MLH1’s recruitment to a DNA…

Group Focuses on Rare Disease Clinical Trial Participation

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 an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…

AMT-130 Gene Therapy Appears Safe; More Data Forthcoming

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 received the low dose of the therapy — which is delivered directly into the brain — and two…

Patients, Caregivers Asked to Help Adira in Choosing Grant Awards

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 most common neurodegenerative diseases — namely, Huntington’s disease, amyotrophic lateral sclerosis, multiple sclerosis, Alzheimer’s disease, and…

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

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 a need for long-term psychological support for people with presymptomatic Huntington’s to help them develop proactive coping strategies and…