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 infants with type 1 SMA aged 2-7mos. and in people with types 2/3 SMA aged 2-25 yrs/ respectively The submission also includes data from JEWELFISH […]Read More
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 of people living with SMA: FIREFISH in symptomatic infants aged 2-7 mos, and SUNFISH in children and adults aged 2-25yrs. The two studies demonstrated improvements […]Read More
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 or point mutations aged < 6mos. at the time of gene therapy The P-III STR1VE study results: alive patients (95%); patients reached 10.5mos. (86%); CHOP-INTEND […]Read More
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 time of first dose aged 6 wks. The study resulted in early treatment allow 100% progressive gains in motor function, reduction in mortality in infants, […]Read More
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