Concordant utrophin upregulation in phenotypically discordant DMD/BMD brothers
Author:
Funder
FAPESP/CEPID
CNPq-INCT
FINEP
AACD
Publisher
Elsevier BV
Subject
Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health
Reference15 articles.
1. Pharmacologically targeting the primary defect and downstream pathology in Duchenne muscular dystrophy;Fairclough;Curr Gene Ther,2012
2. Safety, tolerability, and pharmacokinetics of SMT C1100, a 2-arylbenzoxazole utrophin modulator, following single- and multiple-dose administration to healthy male adult volunteers;Tinsley;J Clin Pharmacol,2015
3. Therapy of genetic disorders – novel therapies for Duchenne muscular dystrophy;Seto;Curr Pediatr Rep,2014
4. Microtubule binding distinguishes dystrophin from utrophin;Belanto;Proc Natl Acad Sci U S A,2014
5. Milder course in Duchenne patients with nonsense mutations and no muscle dystrophin;Zatz;Neuromuscul Disord,2014
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1. Upregulation of utrophin improves the phenotype of Duchenne muscular dystrophy hiPSC-derived CMs;Molecular Therapy - Nucleic Acids;2024-09
2. Phenotypic Spectrum of Dystrophinopathy Due to Duchenne Muscular Dystrophy Exon 2 Duplications;Neurology;2021-12-22
3. Implications of increased S100β and Tau5 proteins in dystrophic nerves of two mdx mouse models for Duchenne muscular dystrophy;Molecular and Cellular Neuroscience;2020-06
4. Molecular Therapies for Muscular Dystrophies;Current Treatment Options in Neurology;2018-06-21
5. Skeletal muscle secretome in Duchenne muscular dystrophy: a pivotal anti-inflammatory role of adiponectin;Cellular and Molecular Life Sciences;2017-02-10
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