Zolgensma, which was FDA approved in May 2019, is administered as an intravenous (IV) infusion, with dosing based on patient weight, in pediatric patients under 2 years of age with SMA caused by...

Pipeline (late-stage development) Name Manufacturer Route of administration Mechanism of action Proposed / studied indication Status Tividenofusp alfa (DNL310) AstraZeneca IV a fusion protein ERT...

Generic Name Brand Name Manufacturer Indication(s) Route of Administration Month Approved* aprocitentan Tryvio® Idosia Difficult-to-control or resistant hypertension Oral March budesonide Eohilia™...

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