Chronic Treatment with the AMPK Agonist AICAR Prevents Skeletal Muscle Pathology but Fails to Improve Clinical Outcome in a Mouse Model of Severe Spinal Muscular Atrophy

SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMA

(2015) Vicki L. McGovern et al.   HUMAN MOLECULAR GENETICS

Low levels of Survival Motor Neuron protein are sufficient for normal muscle function in the SMNΔ7 mouse model of SMA

(2015) Chitra C. Iyer et al.   HUMAN MOLECULAR GENETICS

Resistance strength training exercise in children with spinal muscular atrophy

(2015) Aga Lewelt et al.   MUSCLE & NERVE

Training improves oxidative capacity, but not function, in spinal muscular atrophy type III

(2015) Karen Lindhardt Madsen et al.   MUSCLE & NERVE

Spinal Muscular Atrophy Therapeutics: Where do we Stand?

(2015) Constantin d’Ydewalle et al.   Neurotherapeutics

Spinal muscular atrophy patient-derived motor neurons exhibit hyperexcitability

(2015) Huisheng Liu et al.   Scientific Reports

Assays for the Identification and Prioritization of Drug Candidates for Spinal Muscular Atrophy

(2014) Jonathan J. Cherry et al.   ASSAY AND DRUG DEVELOPMENT TECHNOLOGIES

Myogenic program dysregulation is contributory to disease pathogenesis in spinal muscular atrophy

(2014) Justin G. Boyer et al.   HUMAN MOLECULAR GENETICS

Survival motor neuron protein deficiency impairs myotube formation by altering myogenic gene expression and focal adhesion dynamics

(2014) Katherine V. Bricceno et al.   HUMAN MOLECULAR GENETICS

Requirement of enhanced Survival Motoneuron protein imposed during neuromuscular junction maturation

(2014) Shingo Kariya et al.   JOURNAL OF CLINICAL INVESTIGATION

Mechanisms Involved in Spinal Cord Central Synapse Loss in a Mouse Model of Spinal Muscular Atrophy

(2014) Olga Tarabal et al.   JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY

Skeletal Muscle DNA Damage Precedes Spinal Motor Neuron DNA Damage in a Mouse Model of Spinal Muscular Atrophy (SMA)

(2014) Saniya Fayzullina et al.   PLoS One

Presynaptic Localization of Smn and hnRNP R in Axon Terminals of Embryonic and Postnatal Mouse Motoneurons

(2014) Benjamin Dombert et al.   PLoS One

The therapeutic potential of skeletal muscle plasticity in Duchenne muscular dystrophy: phenotypic modifiers as pharmacologic targets

(2013) Vladimir Ljubicic et al.   FASEB JOURNAL

Splicing therapy for neuromuscular disease

(2013) Andrew G.L. Douglas et al.   MOLECULAR AND CELLULAR NEUROSCIENCE

ROLE of exercise in maintaining the integrity of the neuromuscular junction

(2013) Hiroshi Nishimune et al.   MUSCLE & NERVE

Chronic treatment with lithium does not improve neuromuscular phenotype in a mouse model of severe spinal muscular atrophy

(2013) E. Dachs et al.   NEUROSCIENCE

AMP-activated protein kinase at the nexus of therapeutic skeletal muscle plasticity in Duchenne muscular dystrophy

(2013) Vladimir Ljubicic et al.   TRENDS IN MOLECULAR MEDICINE

Antisense oligonucleotide mediated therapy of spinal muscular atrophy

(2013) Senthilkumar Sivanesan et al.   Translational Neuroscience

More than a bystander: the contributions of intrinsic skeletal muscle defects in motor neuron diseases

(2013) Justin G. Boyer et al.   Frontiers in Physiology

AMPK Activation Stimulates Autophagy and Ameliorates Muscular Dystrophy in the mdx Mouse Diaphragm

(2012) Marion Pauly et al.   AMERICAN JOURNAL OF PATHOLOGY

Severe SMA mice show organ impairment that cannot be rescued by therapy with the HDACi JNJ-26481585

(2012) Julia Schreml et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Antisense-based therapy for the treatment of spinal muscular atrophy

(2012) Frank Rigo et al.   JOURNAL OF CELL BIOLOGY

Preservation of VGLUT1 synapses on ventral calbindin-immunoreactive interneurons and normal locomotor function in a mouse model of spinal muscular atrophy

(2012) Vatsala Thirumalai et al.   JOURNAL OF NEUROPHYSIOLOGY

Motor Neuron Rescue in Spinal Muscular Atrophy Mice Demonstrates That Sensory-Motor Defects Are a Consequence, Not a Cause, of Motor Neuron Dysfunction

(2012) R. G. Gogliotti et al.   JOURNAL OF NEUROSCIENCE

Survival Motor Neuron Protein in Motor Neurons Determines Synaptic Integrity in Spinal Muscular Atrophy

(2012) T. L. Martinez et al.   JOURNAL OF NEUROSCIENCE

Defects in neuromuscular junction remodelling in the Smn2B/− mouse model of spinal muscular atrophy

(2012) Lyndsay M. Murray et al.   NEUROBIOLOGY OF DISEASE

Correction: Muscarinic and Nicotinic Modulation of Thalamo-Prefrontal Cortex Synaptic Pasticity In Vivo

(2012) Lezio Soares Bueno-Junior et al.   PLoS One

Spinal muscular atrophy: going beyond the motor neuron

(2012) Gillian Hamilton et al.   TRENDS IN MOLECULAR MEDICINE

Metabolic remodeling agents show beneficial effects in the dystrophin-deficient mdx mouse model

(2012) Vanessa E Jahnke et al.   Skeletal Muscle

Identification of a Maleimide-Based Glycogen Synthase Kinase-3 (GSK-3) Inhibitor, BIP-135, That Prolongs the Median Survival Time of Δ7 SMA KO Mouse Model of Spinal Muscular Atrophy

(2011) Po C. Chen et al.   ACS Chemical Neuroscience

Chronic AMPK stimulation attenuates adaptive signaling in dystrophic skeletal muscle

(2011) Vladimir Ljubicic et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

Muscles in a mouse model of spinal muscular atrophy show profound defects in neuromuscular development even in the absence of failure in neuromuscular transmission or loss of motor neurons

(2011) Young il Lee et al.   DEVELOPMENTAL BIOLOGY

The contribution of mouse models to understanding the pathogenesis of spinal muscular atrophy

(2011) J. N. Sleigh et al.   Disease Models & Mechanisms

Temporal requirement for high SMN expression in SMA mice

(2011) T. T. Le et al.   HUMAN MOLECULAR GENETICS

Severe neuromuscular denervation of clinically relevant muscles in a mouse model of spinal muscular atrophy

(2011) K. K. Y. Ling et al.   HUMAN MOLECULAR GENETICS

Chronic AMPK activation evokes the slow, oxidative myogenic program and triggers beneficial adaptations in mdx mouse skeletal muscle

(2011) V. Ljubicic et al.   HUMAN MOLECULAR GENETICS

Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy

(2011) Cathleen M. Lutz et al.   JOURNAL OF CLINICAL INVESTIGATION

Defective Neuromuscular Junction Organization and Postnatal Myogenesis in Mice With Severe Spinal Muscular Atrophy

(2011) Elisabet Dachs et al.   JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY

Permanent central synaptic disconnection of proprioceptors after nerve injury and regeneration. I. Loss of VGLUT1/IA synapses on motoneurons

(2011) Francisco J. Alvarez et al.   JOURNAL OF NEUROPHYSIOLOGY

A screen for regulators of survival of motor neuron protein levels

(2011) Nina R Makhortova et al.   Nature Chemical Biology

A critical smn threshold in mice dictates onset of an intermediate spinal muscular atrophy phenotype associated with a distinct neuromuscular junction pathology

(2011) Mélissa Bowerman et al.   NEUROMUSCULAR DISORDERS

Early Functional Impairment of Sensory-Motor Connectivity in a Mouse Model of Spinal Muscular Atrophy

(2011) George Z. Mentis et al.   NEURON

Stem Cell Transplantation for Motor Neuron Disease: Current Approaches and Future Perspectives

(2011) Genevieve Gowing et al.   Neurotherapeutics

Spinal Muscular Atrophy: A Clinical and Research Update

(2011) Jennifer A. Markowitz et al.   PEDIATRIC NEUROLOGY

Fiber Types in Mammalian Skeletal Muscles

(2011) Stefano Schiaffino et al.   PHYSIOLOGICAL REVIEWS

Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

(2011) M. A. Passini et al.   Science Translational Medicine

Stem cell-derived neurotrophic support for the neuromuscular junction in spinal muscular atrophy

(2010) Tanya J Wyatt et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Drug discovery and development for spinal muscular atrophy: lessons from screening approaches and future challenges for clinical development

(2010) Rebecca M Pruss et al.   Future Medicinal Chemistry

Antisense oligonucleotides and spinal muscular atrophy: skipping along

(2010) A. H. M. Burghes et al.   GENES & DEVELOPMENT

Regulation of exercise-induced fiber type transformation, mitochondrial biogenesis, and angiogenesis in skeletal muscle

(2010) Zhen Yan et al.   JOURNAL OF APPLIED PHYSIOLOGY

Reduced Survival of Motor Neuron (SMN) Protein in Motor Neuronal Progenitors Functions Cell Autonomously to Cause Spinal Muscular Atrophy in Model Mice Expressing the Human Centromeric (SMN2) Gene

(2010) G.-H. Park et al.   JOURNAL OF NEUROSCIENCE

Impaired Synaptic Vesicle Release and Immaturity of Neuromuscular Junctions in Spinal Muscular Atrophy Mice

(2009) L. Kong et al.   JOURNAL OF NEUROSCIENCE

AMP-activated protein kinase response to contractions and treatment with the AMPK activator AICAR in young adult and old skeletal muscle

(2009) D.M. Thomson et al.   JOURNAL OF PHYSIOLOGY-LONDON

Increases in nuclear p65 activation in dystrophic skeletal muscle are secondary to increases in the cellular expression of p65 and are not solely produced by increases in IκB-α kinase activity

(2009) Rajvir Singh et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?

(2009) Arthur H. M. Burghes et al.   NATURE REVIEWS NEUROSCIENCE

AMPK and PPARδ Agonists Are Exercise Mimetics

(2008) Vihang A. Narkar et al.   CELL

AMP-activated protein kinase in skeletal muscle: From structure and localization to its role as a master regulator of cellular metabolism

(2008) C. A. Witczak et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Identification of a battery of tests for drug candidate evaluation in the SMNΔ7 neonate model of spinal muscular atrophy

(2008) Bassem F. El-Khodor et al.   EXPERIMENTAL NEUROLOGY

Exercise-Induced Activation of NMDA Receptor Promotes Motor Unit Development and Survival in a Type 2 Spinal Muscular Atrophy Model Mouse

(2008) O. Biondi et al.   JOURNAL OF NEUROSCIENCE