Patients had fewer bleeds after treatment with Beqvez compared to standard treatment, but questions remain about which ...

The FDA expects to issue a decision in the first quarter of 2025, and if the agency grants approval, it will expand the biomarker-eligible population for the drug.

In a draft guidance, the institute did not recommend Orserdu, but is collecting additional data and public comments on this preliminary decision.

The clinical trial testing ATA-200 to treat a type of LGMD caused by mutations in the SGCG gene has already been cleared in Italy and France.