Shots: The FDA decision to lift the partial clinical trial hold of the OAV-101 IT clinical program is based on data from a nonclinical toxicology study in NHP The company […]readmore
Tags : SMA
Shots: The MAA submission is based on dose-finding Part 1 and confirmatory Part 2 of the FIREFISH and SUNFISH studies evaluating the efficacy and safety of Evrysdi (risdiplam) in symptomatic […]readmore
Shots: The US FDA has approved Evrysdi to treat SMA in adults and children ≥ 2mos. The approval is based on two clinical studies designed to represent a broad spectrum […]readmore
Shots: The P-III STR1VE study involves assessing of Zolgensma in 22 patients with SMA Type1 + one or two copies of the SMN2 backup gene + bi-allelic SMN1 gene deletion […]readmore
Shots: The approval is based on P-II NURTURE study results assessing Spinraza in 300 infant patients who were genetically diagnosed with SMA and had not experienced any symptoms by the […]readmore