Results 121 to 130 of about 2,067 (154)

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).
Sheridan M. Hoy (4897468)
openaire   +3 more sources

Safety of Onasemnogene Abeparvovec for Patients With Spinal Muscular Atrophy 8.5 kg or Heavier in a Global Managed Access Program [PDF]

Pediatric Neurology, 2022
BACKGROUND: Spinal muscular atrophy is a rare, neurodegenerative disorder caused by biallelic deletions in the survival motor neuron (SMN1) gene. Onasemnogene abeparvovec is a one-time, intravenous gene replacement therapy designed to deliver the SMN1 ...
Deepa H Chand, Sandra P Reyna
exaly   +2 more sources

Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys [PDF]

Human Gene Therapy, 2022
Intravenous onasemnogene abeparvovec is approved for the treatment of spinal muscular atrophy in children
Mansfield, Keith, Milton, Mark, Tukov, Francis, Penraat, Kelley, Chand, Deepa, Tukov, Francis Fonyuy, Meseck, Emily, Hartmann, Andreas, Francis Fonyuy Tukov, Mark Milton, Deepa Chand, Kelley Penraat, Andreas Hartmann, Keith Mansfield, Emily Meseck   +14 more
exaly   +2 more sources

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Onasemnogene-abeparvovec

Reactions Weekly
Stephanie J. Phelps, Tracy M. Hagemann, Kelley R. Lee   +2 more
  +12 more sources

Combination Therapy with Nusinersen and Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy Type I

Journal of Clinical Medicine, 2021
Background: Spinal muscular atrophy (SMA) is a neuromuscular progressive disease, characterized by decreased amounts of survival motor neuron (SMN) protein, due to an autosomal recessive genetic defect.
Corina Sporea, Elena-Silvia Shelby, Vlad Dima   +2 more
exaly   +2 more sources

Onasemnogene-abeparvovec

Reactions Weekly, 2023
  +7 more sources

Onasemnogene abeparvovec

Reactions Weekly, 2022
  +6 more sources

Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovec [PDF]

European Journal of Paediatric Neurology
Onasemnogene abeparvovec; Spinal muscular atrophy; Survival motor neuron 2 geneOnasemnogene abeparvovec; Atròfia muscular espinal; Gen de la neurona motora 2 de supervivènciaOnasemnogene abeparvovec; Atrofia muscular espinal; Gen de la neurona motora 2 ...
Sharon Aharoni, Laurent Servais, Eduardo Tizzano   +2 more
exaly   +2 more sources