Results 171 to 180 of about 2,700 (197)

Onasemnogene abeparvovec

Reactions Weekly, 2023
  +7 more sources

Onasemnogene-abeparvovec

Reactions Weekly, 2022
  +6 more sources

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.
openaire   +2 more sources

Onasemnogene-abeparvovec

Reactions Weekly, 2021
  +4 more sources

Onasemnogene Abeparvovec: First Global Approval

Drugs, 2019
Onasemnogene abeparvovec (onasemnogene abeparvovec-xioi; formerly AVXS-101; ZOLGENSMA®) is an adeno-associated viral vector-based gene therapy designed to deliver a functional copy of the human survival motor neuron (SMN) gene to the motor neuron cells of patients with spinal muscular atrophy (SMA).
openaire   +2 more sources

Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue

Nature Medicine, 2021
Arthur H M Burghes, Vicki McGovern, Robert F Hevner   +2 more
exaly  

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

Nature Medicine, 2022
Kevin A Strauss, Michelle A Farrar Mbbs, Francesco Muntoni   +2 more
exaly  

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

Lancet Neurology, The, 2021
John W Day, Richard S Finkel, Claudia A Chiriboga   +2 more
exaly  

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial

Nature Medicine, 2022
Kevin A Strauss, Michelle A Farrar Mbbs, Francesco Muntoni   +2 more
exaly  

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial

Lancet Neurology, The, 2021
Eugenio Mercuri, Francesco Muntoni, Giovanni Baranello   +2 more
exaly