This Week in Rare Disease Breakthroughs 🧬 | Pivotal Approvals, New Orphan Drug Designations, AI-Driven Biomarkers and more!🧬
From the NHS approving the first-ever APDS treatment to the FDA’s green light for ZEVASKYN™, the first cell-based gene therapy for RDEB, and the EMA’s recognition of innovation in DMD and AML, this roundup is packed with game-changing milestones.
💊 Highlights include:
🏥 Joenja (leniolisib) approved by the NHS for ultra-rare APDS
🏅 Orphan drug designations for DYNE-251 (DMD) and AB8939 (AML)
⚖️ FDA’s response to Equillium’s Itolizumab for acute GvHD
📊 Promising PSC data from Ipsen’s Elafibranor
🔋 BPM31510 shows targeted CoQ10 delivery in mitochondrial-disease models (BPGbio)
🤖 AI muscle-imaging biomarker tech in FSHD trials from Epicrispr
💡 ZEVASKYN™ gets FDA nod as the first cell-based gene therapy for RDEB
⏳ FDA misses PDUFA date for Stealth BioTherapeutics’ ultra-rare-disease candidate
💰 Merck KGaA acquires SpringWorks for $3.9 billion
Whether you’re a clinician, researcher, or investor, these updates are essential viewing to stay informed on the future of rare-disease care.
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