<img height="1" width="1" style="display:none;" alt="" src="https://px.ads.linkedin.com/collect/?pid=2653956&amp;fmt=gif">
Image Overlay - Rare Disease Treatment Unexpectedly Rejected by the FDA
Image Overlay - Rare Disease Treatment Unexpectedly Rejected by the FDA

Rare Disease Treatment Unexpectedly Rejected by the FDA

September 4, 2019

Rare Disease Treatment Unexpectedly Rejected by the FDA

In a surprise decision, the U.S. Food and Drug Administration has rejected Sarepta’s experimental exon-skipping drug Vyondys 53 (golodirsen) for the treatment of Duchenne muscular dystrophy, citing risks of both infection and kidney toxicity. Yourway is dedicated to supporting clinical trials for advanced therapies for orphan diseases.

 Back to Index
Stay Connected

Want to stay informed about the latest news, updates, and opportunities at Yourway?

Join our newsletter mailing list to never miss an update!

Media

Upcoming Events

Clinical Trial Supply New England

April 14-15, 2026
Boston, MA

GCSG 2026 US Conference

April 27-29, 2026
San Diego, CA

Media

Articles

White-Glove by Design: The Yourway Standard in Clinical Logistics

Open chat
Come chat with us!
Hello! How can I help you?