Professor Dr Eugenio Mercuri

blue and clear capsule with a silver DNA stand inside - idea of gene therapy

Zolgensma^® meaningfully improves spinal muscular atrophy in patients

Asymptomatic and symptomatic children treated with Zolgensma® (onasemnogene abeparvovec) gene…

21 June 2021 | By Hannah Balfour (European Pharmaceutical Review)

Asymptomatic and symptomatic children treated with Zolgensma® (onasemnogene abeparvovec) gene therapy achieved motor milestones never observed in the natural history of spinal muscular atrophy.

DNA strand with cross hares over it - idea of targeting genes for gene therapy

news

SMA drug Zolgensma^® has “significant therapeutic benefit”, according to interim data

Treatment with Zolgensma® (onasemnogene abeparvovec) resulted in rapid and sustained…

5 October 2020 | By Hannah Balfour (European Pharmaceutical Review)

Treatment with Zolgensma® (onasemnogene abeparvovec) resulted in rapid and sustained improvement in motor function in spinal muscular atrophy (SMA) patients.

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Recommended

Professor Dr Eugenio Mercuri

Zolgensma® meaningfully improves spinal muscular atrophy in patients

SMA drug Zolgensma® has “significant therapeutic benefit”, according to interim data

Zolgensma^® meaningfully improves spinal muscular atrophy in patients

SMA drug Zolgensma^® has “significant therapeutic benefit”, according to interim data