Published 13 days ago • loading... • Updated 13 days ago

MDA 2025: SMA gene therapy 'transformational,' per Novartis exec

Summary by SMA News Today

It’s been almost six years since the one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) was first approved in the U.S. to treat young children with spinal muscular atrophy (SMA). Now, more than 95% of babies diagnosed with SMA are being treated with Zolgensma, according to Daniel Grant, vice president and global program head at Novartis, the company that markets the therapy. Since its U.S. approval in May 2019, the SMA gene therapy …

This story is only covered by news sources that have yet to be evaluated by the independent media monitoring agencies we use to assess the quality and reliability of news outlets on our platform. Learn more here.

2 Articles

All

Left

Center

Right

totalwealthresearch.com

Krystal (KRYS) Stock Analysis: Undervalued Gene Therapy Leader Using AI

Remember when gene therapy was the hot topic? How did we forget that we could literally edit human DNA? Well, their loss is our gain. The stock I want to show you today isn’t your average biotech. It’s a $5 billion powerhouse tackling rare diseases. Unlike most biotech companies, it generates positive cash flow. And the best part – it’s incredibly cheap. You’re paying just $25 for every $1 in future profits for a biotech company that leverages A…

13 days ago

Read Full Article