Nonprofit-led gene therapy breaks through with EU backing, changing the rare disease model
einnews.com
Nonprofit-led gene therapy breaks through with EU backing, changing the rare disease model
Gene therapy for CTNNB1 Syndrome advances to trials with EU recognition, preclinical success, and political support for affordable public access.
Apr 2, 2025
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Pathology Navigator on Instagram: "👉 Follow • 💾 Save • 🔁 Share with your study buddy 🧬 MARFAN SYNDROME (Autosomal dominant) • FBN1 (fibrillin-1) mutation • Chromosome 15q21 • Weak connective tissue + ↑ TGF-β signaling ⚙️ 👤 Body build shown in the image • Tall stature, very thin build • Long limbs (dolichostenomelia) • Arachnodactyly ✋ • Pectus excavatum (sunken chest) • Scoliosis curve 👁️ Eye clue (super high-yield) • Ectopia lentis = superotemporal lens displacement 🔍 ✋ Hand sign • Posit
Pathology Navigator on Instagram: "👉 Follow • 💾 Save • 🔁 Share with your study buddy 🧬 MARFAN SYNDROME (Autosomal dominant) • FBN1 (fibrillin-1) mutation • Chromosome 15q21 • Weak connective tissue + ↑ TGF-β signaling ⚙️ 👤 Body build shown in the image • Tall stature, very thin build • Long limbs (dolichostenomelia) • Arachnodactyly ✋ • Pectus excavatum (sunken chest) • Scoliosis curve 👁️ Eye clue (super high-yield) • Ectopia lentis = superotemporal lens displacement 🔍 ✋ Hand sign • Posit
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