The Huntington’s Disease Society of America (HDSA) will contribute nearly $1.9 million to its Centers of Excellence program this year, growing it by one site plus two regional partner sites. These additions bring to 55 the number of grant-funded Centers of Excellence located across the U.S. and to 10…
A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that…
The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…
Spending more time on leisure activities — which contributes to a person’s cognitive reserve, or the ability to improvise — is associated with lesser cognitive and functional decline in people with early manifest Huntington’s disease, a study shows. In addition, a greater number of CAG repeats within the HTT gene…
It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.
The first two patients in Europe have been dosed in uniQure’s Phase 1/2 clinical trial investigating AMT-130, a one-time investigational gene therapy for Huntington’s disease. The trial is underway in Poland, where it is still open for enrollment, and is expected to expand to other sites in the…
Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…
People with Huntington’s disease who have a greater sense of purpose and meaning are more likely to find contentment in life and have better health-related quality of life, regardless of the severity of most of their symptoms. That’s according to the study “Meaning and purpose in…
A motion to expand specialist services for families in Scotland affected by Huntington’s disease (HD) received what’s being hailed as a historic level of cross-party support in the Scottish Parliament. Backed by 98% of members of the Scottish Parliament (MSPs), the call to action is said to be the…
Certain people with Huntington’s disease — specifically, younger adults with milder disease — may have benefited from treatment with tominersen in the Phase 3 GENERATION HD1 clinical trial, according to a new analysis of study data. Based on the results, its developer Roche is planning to launch a Phase 2…
Get regular updates to your inbox.