JCR Pharmaceuticals Wins UAE Approval for IZCARGO
HYOGO, Japan, July 21, 2026 JCR Pharmaceuticals announced that the United Arab Emirates (UAE) has granted marketing authorization for...
enzyme replacement therapy
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Spruce Biosciences Reports Six-Year TA-ERT Data in MPS IIIB
SOUTH SAN FRANCISCO, Calif., June 8, 2026 Spruce Biosciences announced new long-term clinical data demonstrating the potential of tralesinidase...
FDA Approves AVLAYAH for Hunter Syndrome Treatment
SOUTH SAN FRANCISCO, Calif., March 25, 2026 In a landmark regulatory milestone, Denali Therapeutics announced that the U.S. Food...
CHMP Endorses Monthly Elfabrio Dosing for Fabry Disease
PARMA, Italy and CARMIEL, Israel, Jan. 30, 2026 — Chiesi Global Rare Diseases and Protalix BioTherapeutics announced that the...
Immedica Submits NDS for Loargys® (pegzilarginase) in Canada
Stockholm, Sweden – October 31, 2025 — Immedica Pharma has announced that its New Drug Submission (NDS) for Loargys®...
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