This report was first published by Endpoints News. To see the original version, click here
A rare disease gene therapy from Rocket Pharmaceuticals has garnered FDA approval after an earlier rejection for manufacturing problems.
The FDA on Thursday granted accelerated approval to Rocket Pharma’s gene therapy for the severe form of a rare immune disorder called leukocyte adhesion deficiency-I (LAD-I). The gene therapy, to be marketed as Kresladi, was approved to treat certain babies and children with the disease who don’t have a matched sibling stem cell donor.
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