Related references
Note: Only part of the references are listed.The implementation of newborn screening for spinal muscular atrophy: the Australian experience
Didu S. T. Kariyawasam et al.
GENETICS IN MEDICINE (2020)
Neurofilament as a potential biomarker for spinal muscular atrophy
Basil T. Darras et al.
ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY (2019)
Fetal Gene Therapy Using a Single Injection of Recombinant AAV9 Rescued SMA Phenotype in Mice
Afrooz Rashnonejad et al.
MOLECULAR THERAPY (2019)
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study
Darryl C. De Vivo et al.
NEUROMUSCULAR DISORDERS (2019)
Axo-glial interdependence in peripheral nerve development
Robert Fledrich et al.
DEVELOPMENT (2019)
Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment
Daniel M. Ramos et al.
JOURNAL OF CLINICAL INVESTIGATION (2019)
Stasimon Contributes to the Loss of Sensory Synapses and Motor Neuron Death in a Mouse Model of Spinal Muscular Atrophy
Christian M. Simon et al.
CELL REPORTS (2019)
One Year of Newborn Screening for SMA - Results of a German Pilot Project
Katharina Vill et al.
JOURNAL OF NEUROMUSCULAR DISEASES (2019)
Two breakthrough gene-targeted treatments for spinal muscular atrophy: challenges remain
Charlotte J. Sumner et al.
JOURNAL OF CLINICAL INVESTIGATION (2018)
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
E. Mercuri et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)
Hasane Ratni et al.
JOURNAL OF MEDICINAL CHEMISTRY (2018)
Neurofilaments as biomarkers in neurological disorders
Michael Khalil et al.
NATURE REVIEWS NEUROLOGY (2018)
Reduced sensory synaptic excitation impairs motor neuron function via Kv2.1 in spinal muscular atrophy
Emily V. Fletcher et al.
NATURE NEUROSCIENCE (2017)
The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy
Constantin d'Ydewalle et al.
NEURON (2017)
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
J. R. Mendell et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
R. S. Finkel et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Natural history of infantile-onset spinal muscular atrophy
Stephen J. Kolb et al.
ANNALS OF NEUROLOGY (2017)
How Schwann Cells Sort Axons: New Concepts
M. Laura Feltri et al.
NEUROSCIENTIST (2016)
Axon Self-Destruction: New Links among SARM1, MAPKs, and NAD plus Metabolism
Josiah Gerdts et al.
NEURON (2016)
Observational study of spinal muscular atrophy type I and implications for clinical trials
Richard S. Finkel et al.
NEUROLOGY (2014)
SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
Nikolai A. Naryshkin et al.
SCIENCE (2014)
Motor neurons and the generation of spinal motor neuron diversity
Nicolas Stifani
FRONTIERS IN CELLULAR NEUROSCIENCE (2014)
Temporal requirement for SMN in motoneuron development
Le T. Hao et al.
HUMAN MOLECULAR GENETICS (2013)
Survival Motor Neuron Protein in Motor Neurons Determines Synaptic Integrity in Spinal Muscular Atrophy
Tara L. Martinez et al.
JOURNAL OF NEUROSCIENCE (2012)
Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy
Cathleen M. Lutz et al.
JOURNAL OF CLINICAL INVESTIGATION (2011)
Early Functional Impairment of Sensory-Motor Connectivity in a Mouse Model of Spinal Muscular Atrophy
George Z. Mentis et al.
NEURON (2011)
A simple protocol for assessing inter-trial and inter-examiner reliability for two noninvasive measures of limb muscle strength
C. George Carlson et al.
JOURNAL OF NEUROSCIENCE METHODS (2010)
Progenitors of skeletal muscle satellite cells express the muscle determination gene, MyoD
Onur Kanisicak et al.
DEVELOPMENTAL BIOLOGY (2009)
Unmyelinated fibers in human spinal ventral roots: C4 to S2
H-Y Ko et al.
SPINAL CORD (2009)
Astrocyte and muscle-derived secreted factors differentially regulate motoneuron survival
Anna R. Taylor et al.
JOURNAL OF NEUROSCIENCE (2007)
Neuregulin-1 type III determines the ensheathment fate of axons
C Taveggia et al.
NEURON (2005)
Natural history of denervation in SMA:: Relation to age, SMN2 copy number, and function
KJ Swoboda et al.
ANNALS OF NEUROLOGY (2005)
SMNΔ7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN
TT Le et al.
HUMAN MOLECULAR GENETICS (2005)
Axonal neuregulin-1 regulates myelin sheath thickness
GV Michailov et al.
SCIENCE (2004)
Accurate normalization of real-time quantitative RT-PCR data by geometric averaging of multiple internal control genes
Jo Vandesompele et al.
GENOME BIOLOGY (2002)
Cysteine-rich domain isoforms of the neuregulin-1 gene are required for maintenance of peripheral synapses
D Wolpowitz et al.
NEURON (2000)