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

JM Tinsley, RJ Fairclough, R Storer, FJ Wilkes… - PloS one, 2011 - journals.plos.org
Background Duchenne muscular dystrophy (DMD) is a lethal, progressive muscle wasting
disease caused by a loss of sarcolemmal bound dystrophin, which results in the death of the …

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

S Guiraud, SE Squire, B Edwards… - Human molecular …, 2015 - academic.oup.com
Duchenne muscular dystrophy (DMD) is a lethal, X-linked muscle-wasting disease caused
by lack of the cytoskeletal protein dystrophin. There is currently no cure for DMD although …

Utrophin upregulation in Duchenne muscular dystrophy.

RC Hirst, KJ McCullagh, KE Davies - Acta myologica: myopathies …, 2005 - europepmc.org
Duchenne Muscular Dystrophy (DMD) is a devastating, progressive muscle wasting disease
for which there is currently no effective treatment. DMD is caused by mutations in the …

Non-toxic ubiquitous over-expression of utrophin in the mdx mouse

R Fisher, JM Tinsley, SR Phelps, SE Squire… - Neuromuscular …, 2001 - Elsevier
Duchenne muscular dystrophy (DMD) is an inherited, severe muscle wasting disease
caused by the loss of the cytoskeletal protein, dystrophin. Patients usually die in their late …

The potential of utrophin modulators for the treatment of Duchenne muscular dystrophy

S Guiraud, D Roblin, DE Kay - Expert Opinion on Orphan Drugs, 2018 - Taylor & Francis
Introduction: Duchenne muscular dystrophy (DMD) is a lethal, X-linked muscle-wasting
disease caused by the lack of dystrophin. A few years after the identification of the …

Utrophin upregulation for treating Duchenne or Becker muscular dystrophy: how close are we?

P Miura, BJ Jasmin - Trends in molecular medicine, 2006 - cell.com
Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder for which there is
currently no effective treatment. This disorder is caused by mutations or deletions in the …

The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy

S Guiraud, B Edwards, A Babbs… - Human Molecular …, 2019 - academic.oup.com
Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disorder caused by loss of
dystrophin. Several therapeutic modalities are currently in clinical trials but none will achieve …

Drug discovery for Duchenne muscular dystrophy via utrophin promoter activation screening

C Moorwood, O Lozynska, N Suri, AD Napper… - PloS one, 2011 - journals.plos.org
Background Duchenne muscular dystrophy (DMD) is a devastating muscle wasting disease
caused by mutations in dystrophin, a muscle cytoskeletal protein. Utrophin is a homologue …

The role of utrophin in the potential therapy of Duchenne muscular dystrophy

KJ Perkins, KE Davies - Neuromuscular disorders, 2002 - Elsevier
Duchenne muscular dystrophy is an X-linked recessive muscle wasting disease caused by
the absence of the muscle cytoskeletal protein, dystrophin. Dystrophin is a member of the …

Utrophin modulator drugs as potential therapies for Duchenne and Becker muscular dystrophies

P Soblechero‐Martín, A López‐Martínez… - Neuropathology and …, 2021 - Wiley Online Library
Utrophin is an autosomal paralogue of dystrophin, a protein whose deficit causes Duchenne
and Becker muscular dystrophies (DMD/BMD). Utrophin is naturally overexpressed at the …