Results 81 to 86 of about 152 (86)

Onasemnogene Abeparvovec-xioi: Gene Therapy for Spinal Muscular Atrophy

Annals of Pharmacotherapy, 2020
Debra L Stevens, Cheri K Walker
exaly  

Pilocytic astrocytoma in a child with spinal muscular atrophy treated with onasemnogene abeparvovec

Molecular Therapy
Johannes Friese, Philipp John-Neek
exaly  

Continued safety and long-term effectiveness of onasemnogene abeparvovec in Ohio

Neuromuscular Disorders
Megan A Waldrop, Megan Iammarino, Nancy Bass   +2 more
exaly  

Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study

Lancet Regional Health - Europe, The
Johannes Friese, Sabine Illsinger, Oliver Schwartz   +2 more
exaly  

Cost comparison analysis of onasemnogene abeparvovec and nusinersen for treatment of patients with spinal muscular atrophy type 1 in the Netherlands

European Journal of Health Economics
Matthias Bischof, Maarten J Postma, Cornelis Boersma   +2 more
exaly  

Evaluation of cardiac function in patients with SMA after treatment with onasemnogene abeparvovec

Pediatrics International
Satoshi Takasago, Katsuyoshi Shimozawa, Keiji Goishi   +2 more
exaly