An experimental gene therapy was found to slow the progression of Huntington’s disease in a “pivotal” Phase 1/2 study, according to drugmaker uniQure, marking a major step toward a potential first ...
Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
The Nature Index 2025 Research Leaders — previously known as Annual Tables — reveal the leading institutions and countries/territories in the natural and health sciences, according to their output in ...
A new approach to gene therapy for retinitis pigmentosa may help patients with advanced disease regain vision regardless of which genetic mutation is causing the condition. An optogenetic therapy ...
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Japan’s MHLW grants orphan status to Kyowa and Orchard’s MLD therapy
Japan's Ministry of Health, Labor and Welfare (MHLW) has granted orphan regenerative medicine product designation to Kyowa ...
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