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The lead product on AlzeCure’s platform has already demonstrated positive Phase I results.
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Children as young as two with either sickle cell disease or thalassemia are now eligible for the gene therapy.
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The real-world data study tracked Parkinson's disease patients for 12 months during treatment with AbbVie's Vyalev
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Bocunebart's PACAP-targeting mechanism positions it as a potential first-in-class therapy in a thin pipeline of novel late-stage candidates.
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The number of RMAT designations awarded by the FDA increased sharply from 2024, reaching a record of 48 RMAT designations in 2025.
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In the Baby-COMET trial, Nexviazyme met all primary and secondary endpoints, teeing it up for potential label expansion.
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