Onasemnogene abeparvovec - Open Access .click

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Onasemnogene Abeparvovec: A Review in Spinal Muscular Atrophy

CNS Drugs, 2022
Onasemnogene abeparvovec (Zolgensma®) is a gene therapy approved for the treatment of spinal muscular atrophy (SMA). Administered as a one-time intravenous infusion, onasemnogene abeparvovec uses the adeno-associated virus vector to deliver a functional copy of the human survival motor neuron (SMN) gene to motor neuron cells.
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Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy

Journal of Hepatology, 2021
Hugh McMillan, Gerd A Kullak-Ublick, Petra Kaufmann +2 more
exaly

Short-term clinical outcomes of onasemnogene abeparvovec treatment for spinal muscular atrophy

Brain and Development, 2022
Anna Cho, Lee Seungbok, Soo Yeon Kim
exaly

Onasemnogene-abeparvovec/prednisolone

Reactions Weekly, 2022
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Matching-adjusted indirect treatment comparison of onasemnogene abeparvovec and nusinersen for the treatment of symptomatic patients with spinal muscular atrophy type 1

Current Medical Research and Opinion, 2021
Eric Druyts, Matthias Bischof
exaly

Nusinersen/onasemnogene abeparvovec

Reactions Weekly, 2022
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Anakinra/onasemnogene abeparvovec

Reactions Weekly, 2023
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