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Rare Diseases

FDA Approves Filspari to Reduce Proteinuria in FSGS Patients Without Nephrotic Syndrome

The decision is based on a 108-week study where the drug beat irbesartan, giving a new option for a rare kidney disease.

FDA Grants Accelerated Approval to Avlayah for Neurologic Manifestations of Hunter Syndrome

BioMarin to Acquire Amicus for $4.8 Billion in Cash