Gene therapy researchers were converging on a holy grail. A few years ago, researchers at labs and companies reported they had engineered viruses that could ferry corrective genes deep into the brain, giving potential entry to a new world of treatments for Alzheimerβs, Parkinsonβs, and a slew of rare genetic diseases. This summer, after years…
Fondazione Telethon ETS’ Waskyra has become the first gene therapy in the US for patients with Wiskott-Aldrich syndrome (WAS), a rare and life-threatening inherited disease. The Italian non-profit company’s cell-based gene therapy has been cleared by the FDA for use in patients with WAS who are aged six months and older, are healthy enough to…
ASH 2025 had both rain and sun, positive and negative data in Orlando, Fla.ASH 2025 was in Orlando, Fla. Adam Feuerstein / STAT Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The…
CDSCO Panel Approves Akums’ Proposal to Manufacture, Market Nitisinone Dispersible Tablets for Hereditary Tyrosinemia Type 1 We use cookies for analytics, advertising and to improve our site. You agree to our use of cookies by continuing to use our site. To know more, see our Cookie Policy and Cookie Settings.Ok Source link
Delhi HC forms panel to monitor crowdfunding for rare disease treatment We use cookies for analytics, advertising and to improve our site. You agree to our use of cookies by continuing to use our site. To know more, see our Cookie Policy and Cookie Settings.Ok Source link
In the latest tussle over access to medicines, an Indian court has upheld a ruling that found it would be in the public interest to allow a generic company to sell a lower-cost version of a pricey rare disease drug sold by Roche. And the closely watched case is being hailed by patient advocates as…
After months of uncertainty, the Food and Drug Administration offered accelerated approval for an ultra-rare disease drug that had become a symbol of the fraught balancing act between upholding regulatory standards and accommodating desperate patients and their families. The agency endorsed a treatment for Barth syndrome, a rare illness that afflicts about 150 people in…
Four new drugs for rare diseases on anvil, 5 medications for 7 ailments in mkt: VK Paul We use cookies for analytics, advertising and to improve our site. You agree to our use of cookies by continuing to use our site. To know more, see our Cookie Policy and Cookie Settings.Ok Source link
PLI scheme brings down cost of rare disease treatment from crores to lakhs We use cookies for analytics, advertising and to improve our site. You agree to our use of cookies by continuing to use our site. To know more, see our Cookie Policy and Cookie Settings.Ok Source link
In the latest twist over the fate of an ultra-rare disease drug, Stealth BioTherapeutics said it received βconflictingβ signals from the U.S. Food and Drug Administration about information needed to approve its therapy and, consequently, is making contingency plans to close the company. At issue is a treatment for Barth syndrome, a rare illness that…