Tuesday, March 19, 2024
ISSN 2765-8767
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Genetic therapy falls short for Huntington’s Disease

Two pharmaceutical companies have halted clinical trials of gene-targeting therapies for Huntington’s disease (HD), following the drugs’ disappointing performance.

Researchers had hoped that the treatments—known as antisense oligonucleotides (ASOs)—would be a game changer for HD, an incurable genetic condition that affects cognition, behaviour and movement. But back-to-back announcements from Roche, headquartered in Basel, Switzerland, and Wave Life Sciences, in Cambridge, Massachusetts, have dealt a crushing blow to those affected by the disease.

“I was really shocked, really tearful,” says Marion, a woman in London with HD, who was part of one of the trials. “We didn’t see it coming at all. I felt really frightened and worried about my future.” Marion requested that her last name be withheld to protect her privacy.

Source: Scientific American

Daily Remedy

Daily Remedy

Dr. Jay K Joshi serves as the editor-in-chief of Daily Remedy. He is a serial entrepreneur and sought after thought-leader for matters related to healthcare innovation and medical jurisprudence. He has published articles on a variety of healthcare topics in both peer-reviewed journals and trade publications. His legal writings include amicus curiae briefs prepared for prominent federal healthcare cases.

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