April 15th 2022
Shephard Mpofu, MD, part of the Novartis Gene Therapies leadership, shared his thoughts on the latest phase 3 data on onasemnogene abeparvovec (Zolgensma) from the SPR1NT trial.
March 16th 2022
Data presented at MDA 2022 show improvements in motor and pulmonary function and patient-reported outcomes.
Sarepta also presented updated data from Study 101 of SRP-9001 at MDA 2022.
March 15th 2022
New data presented at MDA 2022 showcased positive motor and bulbar function data.
March 19th 2021
SGT-001 elicited promising improvements in functional and biomarker end points for patients with Duchenne muscular dystrophy.
March 18th 2021
SRP-9003 elicited sustained protein expression in muscle tissue and stabilized North Star Assessment for Dysferlinopathies scores at 2 years in patients with limb-girdle muscular dystrophy Type 2E.
Updated findings from across a number of studies highlighted the significant benefits of onasemnogene abeparvovec for children with spinal muscular atrophy.