Impressive efficacy of Spinraza administered presymptomatically
Impressive! The results of NURTURE, a presymptomatic clinical trial of Spinraza, are out. The drug appears to prevent SMA altogether in the majority of children when started early!
Category: Updates
Zolgensma shows impressive results in older children when administered by intrathecal injection
A gene therapy approved for treating spinal muscular atrophy in infants has also shown improved efficacy in older children.
Impressive efficacy of Spinraza administered presymptomatically
Impressive! The results of NURTURE, a presymptomatic clinical trial of Spinraza, are out. The drug appears to prevent SMA altogether in the majority of children when started early!
Zolgensma shows impressive results in older children when administered by intrathecal injection
A gene therapy approved for treating spinal muscular atrophy in infants has also shown improved efficacy in older children.
Roche presents new data on risdiplam
Roche has presented newest data on safety and efficacy of risdiplam, the next drug being developed to treat all forms of spinal muscular atrophy. The data reinforces earlier observations that risdiplam appears to be safe and effective across the full spectrum of the disease. The company also announced the dates of regulatory submission in the…
Roche presents new data on risdiplam
Roche has presented newest data on safety and efficacy of risdiplam, the next drug being developed to treat all forms of spinal muscular atrophy. The data reinforces earlier observations that risdiplam appears to be safe and effective across the full spectrum of the disease. The company also announced the dates of regulatory submission in the…
Sam McKie gets his first Spinraza injection
Friday the 4th of October will be remembered for a very long time. It was the day that ended 2 and a 1/2 years of campaigning and fighting for access to Spinraza for Sam.
Sam McKie gets his first Spinraza injection
Friday the 4th of October will be remembered for a very long time. It was the day that ended 2 and a 1/2 years of campaigning and fighting for access to Spinraza for Sam.