🔬 Discover the latest breakthroughs in rare disease research from this week’s global biotech and regulatory landscape!
In this edition, we cover exciting FDA orphan drug designations, innovative gene therapies, major funding rounds, and more—each one advancing the treatment landscape for ultra-rare diseases. From promising treatments for MPS IIIB to gene therapies for NEDAMSS, this roundup is packed with transformative milestones shaping the future of rare disease care.
🚨 Don’t miss these major developments:
💥 FDA grants Orphan Drug Designation to JR-446 for MPS IIIB (Sanfilippo Syndrome Type B)
🏅 Infinant Health receives FDA Orphan Drug & Rare Pediatric Disease Designations for NEC in preterm infants
🤔 FDA grants Orphan Drug Designation to THE001 for soft tissue sarcomas
🌍 Biodexa’s eRapa gets Orphan Drug Designation in the EU for Familial Adenomatous Polyposis (FAP)
💉 Andelyn Biosciences delivers gene therapy for rare NEDAMSS disease with promising early results
📈 Ultragenyx presents its rare disease strategy and projected $640M–$670M revenue for 2025
💰 Azafaros raises €132M ($150M) for rare neuro-metabolic diseases
🩸 Takeda’s Adzynma gets approval from MHRA for congenital thrombotic thrombocytopenic purpura (cTTP) in the UK
📢 Stay Ahead in Rare Disease Research!
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