Implications of circulating neurofilaments for spinal muscular atrophy treatment early in life: A case series
Open Access
- 30 October 2021
- journal article
- research article
- Published by Elsevier in Molecular Therapy - Methods & Clinical Development
- Vol. 23, 524-538
Abstract
No abstract availableKeywords
Funding Information
- NIH
- National Institute of Neurological Disorders and Stroke (R21NS108015)
- Biogen
This publication has 33 references indexed in Scilit:
- Evaluation of Peripheral Blood Mononuclear Cell Processing and Analysis for Survival Motor Neuron ProteinPLOS ONE, 2012
- Candidate Proteins, Metabolites and Transcripts in the Biomarkers for Spinal Muscular Atrophy (BforSMA) Clinical StudyPLOS ONE, 2012
- Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric DisordersMolecular Therapy, 2011
- Validation of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)Pediatric Physical Therapy, 2011
- Compound muscle action potential and motor function in children with spinal muscular atrophyMuscle & Nerve, 2010
- The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): Test development and reliabilityNeuromuscular Disorders, 2010
- A Positive Modifier of Spinal Muscular Atrophy in the SMN2 GeneThe American Journal of Human Genetics, 2009
- An expanded version of the Hammersmith Functional Motor Scale for SMA II and III patientsNeuromuscular Disorders, 2007
- Natural history of denervation in SMA: Relation to age, SMN2 copy number, and functionAnnals of Neurology, 2005
- Neurofilament heavy chain side arm phosphorylation regulates axonal transport of neurofilamentsJournal of Cell Biology, 2003