Second-generation compound for the modulation of utrophin in the therapy of DMD

Safety, tolerability, and pharmacokinetics of SMT C1100, a 2-arylbenzoxazole utrophin modulator, following single- and multiple-dose administration to healthy male adult volunteers

(2015) Jon Tinsley et al.   JOURNAL OF CLINICAL PHARMACOLOGY

A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

(2015) Jerry R Mendell et al.   MOLECULAR THERAPY

Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

(2015) Aurélie Goyenvalle et al.   NATURE MEDICINE

Studying the role of dystrophin-associated proteins in influencing Becker muscular dystrophy disease severity

(2015) J.C. van den Bergen et al.   NEUROMUSCULAR DISORDERS

Flexibility in the N-terminal actin-binding domain: Clues fromin silicomutations and molecular dynamics

(2015) Devlina Chakravarty et al.   PROTEINS-STRUCTURE FUNCTION AND BIOINFORMATICS

Utrophin A is essential in mediating the functional adaptations of mdx mouse muscle following chronic AMPK activation

(2014) H. Al-Rewashdy et al.   HUMAN MOLECULAR GENETICS

Utrophin regulates modal gating of mechanosensitive ion channels in dystrophic skeletal muscle

(2014) Nhi Tan et al.   JOURNAL OF PHYSIOLOGY-LONDON

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

(2014) Thomas Voit et al.   LANCET NEUROLOGY

Arginine butyrate per os protects mdx mice against cardiomyopathy, kyphosis and changes in axonal excitability

(2014) Sara Vianello et al.   NEUROBIOLOGY OF DISEASE

PDE5 inhibition alleviates functional muscle ischemia in boys with Duchenne muscular dystrophy

(2014) M. D. Nelson et al.   NEUROLOGY

Microtubule binding distinguishes dystrophin from utrophin

(2014) J. J. Belanto et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Ex Vivo Gene Editing of the Dystrophin Gene in Muscle Stem Cells Mediated by Peptide Nucleic Acid Single Stranded Oligodeoxynucleotides Induces Stable Expression of Dystrophin in a Mouse Model for Duchenne Muscular Dystrophy

(2014) Farnoosh Nik-Ahd et al.   STEM CELLS

Muscle Structure Influences Utrophin Expression in mdx Mice

(2014) Glen B. Banks et al.   PLoS Genetics

VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects

(2013) Christopher R. Heier et al.   EMBO Molecular Medicine

Identification of FHL1 as a therapeutic target for Duchenne muscular dystrophy

(2013) Colleen E. D'Arcy et al.   HUMAN MOLECULAR GENETICS

Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

(2013) Richard S. Finkel et al.   PLoS One

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

Nitric oxide signalling pathway in Duchenne muscular dystrophy mice: up-regulation of L-arginine transporters

(2012) Jayalakshmi Ramachandran et al.   BIOCHEMICAL JOURNAL

Increased Resting Intracellular Calcium Modulates NF-κB-dependent Inducible Nitric-oxide Synthase Gene Expression in DystrophicmdxSkeletal Myotubes

(2012) Francisco Altamirano et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Successful Regional Delivery and Long-term Expression of a Dystrophin Gene in Canine Muscular Dystrophy: A Preclinical Model for Human Therapies

(2012) Zejing Wang et al.   MOLECULAR THERAPY

Fiji: an open-source platform for biological-image analysis

(2012) Johannes Schindelin et al.   NATURE METHODS

Discovery of 2-Arylbenzoxazoles as Upregulators of Utrophin Production for the Treatment of Duchenne Muscular Dystrophy

(2011) Daniel R. Chancellor et al.   JOURNAL OF MEDICINAL CHEMISTRY

Daily Treatment with SMTC1100, a Novel Small Molecule Utrophin Upregulator, Dramatically Reduces the Dystrophic Symptoms in the mdx Mouse

(2011) Jonathon M. Tinsley et al.   PLoS One

Diaphragm rescue alone prevents heart dysfunction in dystrophic mice

(2010) Alastair Crisp et al.   HUMAN MOLECULAR GENETICS

Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin

(2010) D. Li et al.   JOURNAL OF CELL SCIENCE

Voluntary exercise induces structural remodeling in the hearts of dystrophin-deficient mice

(2010) Jeffrey M. Costas et al.   MUSCLE & NERVE

Biglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in mdx mice

(2010) A. R. Amenta et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Calcium influx is sufficient to induce muscular dystrophy through a TRPC-dependent mechanism

(2009) Douglas P. Millay et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Functional Substitution by TAT-Utrophin in Dystrophin-Deficient Mice

(2009) Kevin J. Sonnemann et al.   PLOS MEDICINE

Effects of UCP3 genotype, temperature and muscle type on energy turnover of resting mouse skeletal muscle

(2008) C. J. Barclay et al.   PFLUGERS ARCHIV-EUROPEAN JOURNAL OF PHYSIOLOGY