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.
News
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…
Annexon’s investigational therapy ANX005 is showing tolerability and a potential to treat people in the earlier stages of Huntington’s disease or at genetic risk of the disorder, according to interim data from an ongoing Phase 2 trial. ANX005, an antibody designed to inhibit the activation of the complement cascade —…
People with Huntington’s disease are more likely to take Austedo (deutetrabenazine) as directed, compared with the similar medication Xenazine (tetrabenazine), according to a new study. The study also found lower discontinuation rates with Austedo, which may suggest that, between these two treatments for chorea, Austedo tends to…
Throughout 2021, Huntington’s Disease News brought you daily coverage of the latest scientific findings, treatment developments, and clinical trials related to Huntington’s disease. As a reminder of what mattered most to you last year, here are the top 10 most-read articles of 2021 with a brief description of what made…
The experimental oral medication branaplam (LMI070) has been granted fast track designation by the U.S. Food and Drug Administration (FDA) as a potential treatment for Huntington’s disease, according to an announcement from the therapy’s developer, Novartis. This designation is designed to speed the development and review of…
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