B. Maru

Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

S. Al-Zaidy, A. Simon Pickard, Kavitha Kotha et al. · Pediatric Pulmonology · 2018 · 219 citations · Full text

Medicine, Genetics, Pediatrics +13

Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients

Daniel C. Malone, Rebecca Dean, R. Arjunji et al. · Journal of Market Access & Health Policy · 2019 · 95 citations · Full text

Pharmacy, Biostatistics, Pharmacoeconomics +17

Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1

Omar Dabbous, B. Maru, Jeroen P. Jansen et al. · Advances in Therapy · 2019 · 93 citations · Full text

Medicine, Neurology, Pediatrics +13

An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER)

Rebecca Dean, I. Jensen, Phil Cyr et al. · Journal of Market Access & Health Policy · 2021 · 53 citations · Full text

Pharmacy, Pharmacoeconomics, Pharmacotherapy +17

Severe communication delays are independent of seizure burden and persist despite contemporary treatments in <i>SCN1A</i>+ Dravet syndrome: Insights from the <scp>ENVISION</scp> natural history study

Μ. Scott Perry, Ingrid E. Scheffer, Joseph Sullivan et al. · Epilepsia · 2023 · 24 citations · Full text

Education, Neurology, Neuroscience +17