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 “conflicting” 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…