January 19, 2018
Cure SMA and our partners in the SMA Industry Collaboration are pleased to announce the release of the Spinal Muscular Atrophy Voice of the Patient (VoP) Report. This report is a thorough written account...
January 17, 2018
We’ve recently released an update to the SMA drug pipeline. This latest version includes:
- 16 active programs, including one approved therapy.
- 14 pharmaceutical partners.
- 6 programs in clinical trials.
- An ever-increasing breadth of...
January 16, 2018
AveXis, a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today provided an overview of the expanded clinical development program for the company’s initial gene therapy candidate, AVXS-101, for...