From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1
Author:
Funder
AveXis, Inc.
Publisher
Elsevier BV
Subject
Clinical Neurology,Developmental Neuroscience,Neurology,Pediatrics, Perinatology, and Child Health
Reference55 articles.
1. Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?;Burghes;Nat Rev Neurosci,2009
2. Quantitative analyses of SMN1 and SMN2 based on real-time lightCycler PCR: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy;Feldkotter;Am J Hum Genet,2002
3. Developmental milestones in type I spinal muscular atrophy;De Sanctis;Neuromuscul Disord,2016
4. Observational study of spinal muscular atrophy type I and implications for clinical trials;Finkel;Neurology,2014
5. Diagnosis and management of spinal muscular atrophy: part 2: pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics;Finkel;Neuromuscul Disord,2018
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