New Rare Disease Drugs and Research Advancements
XTalks
FEBRUARY 27, 2024
Recent FDA Approvals of New Rare Disease Drugs Filspari (sparsentan) for primary immunoglobulin A nephropathy (IgAN), a rare kidney disease, marking it as the first non-immunosuppressive therapy for the condition. Sarepta hopes to clarify Elvidys’ effectiveness in older children in a confirmatory trial.
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