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Trending in Rare Diseases: European Approval for C3G Treatment and more
Автор: LucidQuest
Загружено: 2026-02-01
Просмотров: 1
Описание: Sobi’s Aspaveli® (pegcetacoplan) receives European Commission approval for treating C3G and IC-MPGN, marking a milestone in rare kidney disease therapy. Atossa Therapeutics gains FDA Orphan Drug Designation for (Z)-endoxifen in Duchenne Muscular Dystrophy, accelerating clinical development. Tanabe Pharma announces promising Phase 3 results for MT-7117 in EPP and XLP. Meanwhile, IntraBio’s AQNEURSA® is approved in the EU for Niemann-Pick Type C, and Quoin Pharmaceuticals applies for Orphan Drug Designation for QRX003 in Netherton Syndrome. Key strides in rare disease treatments continue.
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