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Onasemnogene abeparvovec gene therapy for spinal muscular atrophy: A cohort study from the United Arab Emirates

Muscle and Nerve
Vivek Mundada
exaly  

Eculizumab/onasemnogene-abeparvovec

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openaire   +1 more source

Methylprednisolone/Onasemnogene abeparvovec

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Methylprednisolone/onasemnogene abeparvovec/prednisolone

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gene therapy
nusinersen
humans
sma
zolgensma
risdiplam
3. good health
muscular atrophy, spinal
newborn screening
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