Rare Diseases

MeiraGTx to Present 3-Year AAV-hAQP1 Gene Therapy Data for Radiation-Induced Xerostomia Treatment

2 min

FDA Approves RARE-Cure’s Orphan Drug Enzyme Replacement Therapy

FDA · Rare Metabolic Disorders · 5 min

FDA Approves Kresladi: First Gene Therapy for Rare Immune Disorder LAD-I Marks Medical Breakthrough

FDA · Rare genetic immunodeficiency disorders · 3 min

FDA Approves Imfinzi: Breakthrough for Stage III NSCLC Treatment

FDA · Non-Small Cell Lung Cancer · 4 min

FDA Approves Wainua: AstraZeneca's Eplontersen for hATTR-PN

FDA · Hereditary transthyretin-mediated amyloid polyneuropathy · 4 min

FDA Approves Augtyro: Bristol Myers Squibb’s Repotrectinib for ROS1-NSCLC

FDA · Non-Small Cell Lung Cancer · 4 min

Enzyme Replacement Therapy Gaucher: FDA Insights on Efficacy & Safety

FDA · Rare Diseases · 5 min

Gene Therapies for SMA: FDA Market Analysis & Future Outlook 2024

FDA · neuromuscular disorders · 10 min

FDA Grants Priority Review to AstraZeneca's Wilson Disease Treatment

FDA · Rare Diseases · 4 min