This Week in Rare Disease Breakthroughs 🧬 | Gene Therapies, Drug Approvals & Global Policy Shifts
From China’s first gene therapy approval for hemophilia B to Poland’s urgent call for EU-wide reform in rare disease care, this week’s roundup is packed with developments you won’t want to miss.
🔍 Key Highlights:
- Imbria secures $57.5M to accelerate its nHCM heart drug with a new CEO at the helm
- China approves BBM-H901, marking a historic milestone in gene therapy
- Pfizer exits hemophilia gene therapy, while CSL stands by Hemgenix amid access hurdles
- QTORIN™ gel shows 100% patient improvement in a rare pediatric skin condition
- FDA greenlights self-injection option for VYVGART Hytrulo in gMG and CIDP
- Folia and Novartis launch a digital, real-world C3G kidney disease study
- Pretzel Therapeutics publishes a game-changing mtDNA drug discovery in Nature
- Poland pushes for a unified EU strategy to close rare disease treatment gaps
💡 Whether you’re a researcher, clinician, or healthcare strategist, these insights offer a clear view of where rare disease innovation is headed—clinically, commercially, and globally.
📢 Stay Ahead in Rare Disease Research!
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