🧬 This Week’s Must-Know Rare Disease Updates
Catch up on the biggest developments in rare disease—from approvals to designations and long-term data insights:
💊 Scinai Acquires Pincell to Advance Severe Dermatology Antibody
✅ FDA Approves Soleno’s VYKAT XR for Hyperphagia in Prader-Willi Syndrome
🧠 Alexion Showcases Robust Long-Term Data in Rare Neurology at AAN 2025
🧪 LIVMARLI Approved in Japan for Rare Cholestatic Liver Diseases
🧬 Palatin’s Oral MC4R Agonist Gets Orphan Nod for Rare Genetic Obesity
🌍 Crinetics’ Paltusotine Advances in EU With EMA Validation & Orphan Designation
🚨 EMPAVELI Gets FDA Priority Review for C3G and IC-MPGN
🌐 Pharvaris’ Deucrictibant Receives EU Orphan Status for Bradykinin-Mediated Angioedema
📢 Stay Ahead in Rare Disease Research!
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