NEJM This Week NEJM This Week — June 12, 2025
11 snips
Jun 11, 2025 Discover breakthroughs in treatments for pulmonary fibrosis, obesity, and hemophilia B, emphasizing gene editing's role in rare diseases. Delve into the complexities of personalized medicine and the ethics surrounding gene therapy. Explore a gripping case of granulomatosis linked to puzzling ear symptoms and the pressing issue of health disparities affecting Black women in maternal care. The podcast also highlights the importance of advocacy and trust in navigating systemic inequities in healthcare.
AI Snips
Chapters
Transcript
Episode notes
Near-Endomalast Slows Pulmonary Fibrosis
- Near-endomalast significantly slows idiopathic pulmonary fibrosis progression and is also effective in other progressive pulmonary fibrosis types.
- Diarrhea is the main adverse effect, highlighting the need for balancing efficacy and tolerability.
Masdutide Benefits Weight and Health
- Masdutide, a dual GLP-1 and glucagon receptor agonist, provides clinically meaningful weight loss in Chinese adults with overweight or obesity.
- It also improves cardiometabolic risk markers beyond weight reduction alone.
Gene Therapy Durable in Hemophilia B
- A single intravenous dose of AAV gene therapy provides durable factor IX expression in severe hemophilia B.
- This treatment shows long-term efficacy and safety up to 13 years.