FDA grants Ultragenyx's Genglycos accelerated approval, its first gene therapy, for GSDIa in patients 8 and up; list price $2.7M.
Ultragenyx has secured its first approved gene therapy, with the FDA clearing its asset for an ultra-rare disease that can cause life-threatening blood sugar crashes.
…
Get free access to a limited number of articles, plus choose newsletters to get straight to your inbox.
The FDA has granted accelerated approval of a gene therapy to treat children and adults aged at least 8 years with glycogen…
Ultragenyx shares rise after FDA approval of Genglycos, with a $2.7 million wholesale cost and $362 million peak sales estimate.
Patients with GSDIa need to constantly eat cornstarch to avoid dangerous drops in blood sugar
If at first you don’t succeed in gene therapy, then try, try again. | If at first you don’t succeed in gene therapy, then try,…
Grace Science CEO Matt Wilsey says FDA demands could kill its gene therapy for the rare and fatal NGLY1 deficiency by September…
The FDA has reversed course and allowed uniQure to seek accelerated approval for the Huntington's gene therapy AMT-130 after…
