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Journal of Market Access & Health Policy
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March 12, 2021
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, Ivar Jensen, Phil Cyr, et al.
Journal of Child Neurology
|
October 9, 2010
Muscle volume estimation by magnetic resonance imaging in spinal muscular atrophy
Douglas M Sproule, Mark Punyanitya, Wei Shen, et al.
Muscle & Nerve
|
July 30, 2013
The motor neuron response to SMN1 deficiency in spinal muscular atrophy
Peter B Kang, Clifton L Gooch, Michael P McDermott, et al.
Neuromuscular Disorders : NMD
|
July 9, 2010
Adiposity is increased among high-functioning, non-ambulatory patients with spinal muscular atrophy
Douglas M Sproule, Jacqueline Montes, Sally Dunaway, et al.
Pediatric Pulmonology
|
December 15, 2018
Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy
Samiah Al-Zaidy, A Simon Pickard, Kavitha Kotha, et al.
Pediatric Neurology
|
July 7, 2019
Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy
Linda P Lowes, Lindsay N Alfano, W David Arnold, et al.
Journal of Neuromuscular Diseases
|
August 6, 2019
AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort
Samiah A Al-Zaidy, Stephen J Kolb, Linda Lowes, et al.
Nature Medicine
|
October 5, 2021
Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue
Gretchen Thomsen, Arthur H M Burghes, Caroline Hsieh, et al.
Plos One
|
April 9, 2013
SMA-MAP: a plasma protein panel for spinal muscular atrophy
Dione T Kobayashi, Jing Shi, Laurie Stephen, et al.
Neurology
|
August 1, 2014
Observational study of spinal muscular atrophy type I and implications for clinical trials
Richard S Finkel, Michael P McDermott, Petra Kaufmann, et al.
Page
of 4
Search research articles
Search
Showing results (21-30 of 33) with videos related to
Sort By:
Page
of 4
Journal of Market Access & Health Policy
|
March 12, 2021
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, Ivar Jensen, Phil Cyr, et al.
Journal of Child Neurology
|
October 9, 2010
Muscle volume estimation by magnetic resonance imaging in spinal muscular atrophy
Douglas M Sproule, Mark Punyanitya, Wei Shen, et al.
Muscle & Nerve
|
July 30, 2013
The motor neuron response to SMN1 deficiency in spinal muscular atrophy
Peter B Kang, Clifton L Gooch, Michael P McDermott, et al.
Neuromuscular Disorders : NMD
|
July 9, 2010
Adiposity is increased among high-functioning, non-ambulatory patients with spinal muscular atrophy
Douglas M Sproule, Jacqueline Montes, Sally Dunaway, et al.
Pediatric Pulmonology
|
December 15, 2018
Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy
Samiah Al-Zaidy, A Simon Pickard, Kavitha Kotha, et al.
Pediatric Neurology
|
July 7, 2019
Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy
Linda P Lowes, Lindsay N Alfano, W David Arnold, et al.
Journal of Neuromuscular Diseases
|
August 6, 2019
AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort
Samiah A Al-Zaidy, Stephen J Kolb, Linda Lowes, et al.
Nature Medicine
|
October 5, 2021
Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue
Gretchen Thomsen, Arthur H M Burghes, Caroline Hsieh, et al.
Plos One
|
April 9, 2013
SMA-MAP: a plasma protein panel for spinal muscular atrophy
Dione T Kobayashi, Jing Shi, Laurie Stephen, et al.
Neurology
|
August 1, 2014
Observational study of spinal muscular atrophy type I and implications for clinical trials
Richard S Finkel, Michael P McDermott, Petra Kaufmann, et al.
Page
of 4