FDA and multiple outlets highlight approvals including a first gene therapy for severe LAD-1 and other rare-disease drugs, underscoring continued regulatory throughput for ultra-rare conditions.
FDA approved two new drugs for rare diseases this week!
FDA approves Rocket gene therapy for rare immune disorder
The FDA approved a gene therapy for severe leukocyte adhesion deficiency type 1 (LAD-1), an ultra-rare disease
This finding is one of many signals tracked across Healthcare. The live feed updates every few hours with new expert voices, debates, and emerging ideas.
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