Logo

Novartis Publishes Results from Two and Three-Copy Cohorts of Zolgensma in P-III (SPR1NT) Trial for Spinal Muscular Atrophy in the Nature Medicine

Share this
Novartis Publishes Results from Two and Three-Copy Cohorts of Zolgensma in P-III (SPR1NT) Trial for Spinal Muscular Atrophy in the Nature Medicine

Novartis Publishes Results from Two and Three-Copy Cohorts of Zolgensma in P-III (SPR1NT) Trial for Spinal Muscular Atrophy in the Nature Medicine

Shots:

  • The P-III (SPR1NT) trial evaluates Zolgensma in patients with SMA & 2 or 3 copies of SMN2. The trial reinforces the transformational benefit of early treatment with one-time gene therapy
  • The results showed that all children treated symptomatically in both copy cohorts met the 1EPs of sitting alone for ≥30 sec. & standing alone for ≥3 sec. with 79% & 93% achieved milestones within the WHO window of normal development. In the two-copy cohort, patients were able to stand & walk independently
  • In the three-copy cohort, 93% were walked alone with 73% within the WHO window of normal development, AEs were consistent with prior data with no new safety signals, and all children were free of respiratory, nutritional support & serious TRAEs

Ref: Novartis| Image:Novartis 

Click here to­ read the full press release 

Neha

Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.

Share this article on WhatsApp, LinkedIn and Twitter

Join the PharmaShots family of 12000+ subscribers

I accept the Terms and Conditions