Stealth Bio’s Forzinity finally approved as first Barth syndrome therapy
The accelerated approval follows delays and rejections for Stealth Bio’s mitochondria-targeting injection.
22 September 2025
22 September 2025
The accelerated approval follows delays and rejections for Stealth Bio’s mitochondria-targeting injection.
The 2024–28 VPAG is the UK's main cost-containment mechanism on National Health Service (NHS) drug spending.
Tryngolza is designed to reduce the production of apoC-III, a protein involved in triglyceride metabolism.
The EU Data Act will shift data control away from manufacturers and large cloud providers to users of connected products and services and smaller EU companies.
The treatment demonstrated sustained efficacy over a two-year follow-up period.
Armed with fresh capital and new leadership, Genetix sharpens its gene therapy focus.
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