Novartis Reports Updated Efficacy Data of Zolgensma from the P-IIIb (SMART) Study for the Treatment of Spinal Muscular Atrophy (SMA)
Shots:
- The P-IIIb (SMART) trial determines the safety, tolerability & efficacy of zolgensma (IV) in patients (n=24; 8.5kg to 21kg; ~18mos. to 9yrs. of age) with symptomatic SMA having bi-allelic mutations in the SMN1 gene & any copy no. of the SMN2 gene
- In the study, 21/24 patients experienced increased transaminases, 17/24 had transient thrombocytopenia & mean increase in RULM & HFMSE from baseline at wk. 52 was 2.0 points & 3.7 points, respectively
- In the final analysis, majority of patients showed maintained/improved motor milestones, 23/24 patients achieved sitting with slight support, 3 of them achieved standing with support & 1 achieved walking with support at wk. 52. All 6 patients who could walk at baseline maintained it until the end of the study
Ref: Novartis | Image: Novartis
PharmaShots! Your go-to media platform for customized news ranging for multiple indications. For more information connect with us at connect@pharmashots.com
Click here to read the full press release
Tags
Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
