Pseudoexon activation increases phenotype severity in a Becker muscular dystrophy patient

Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Update on the Treatment of Duchenne Muscular Dystrophy

(2013) Louise R. Rodino-Klapac et al.   Current Neurology and Neuroscience Reports

Dystrophin levels and clinical severity in Becker muscular dystrophy patients

(2013) J. C. van den Bergen et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Antisense suppression of donor splice site mutations in the dystrophin gene transcript

(2013) Sue Fletcher et al.   Molecular Genetics & Genomic Medicine

Antisense Oligonucleotide-Mediated Exon Skipping for Duchenne Muscular Dystrophy: Progress and Challenges

(2012) Virginia Arechavala-Gomeza et al.   CURRENT GENE THERAPY

RNA Splicing Manipulation: Strategies to Modify Gene Expression for a Variety of Therapeutic Outcomes

(2011) Steve D. Wilton et al.   CURRENT GENE THERAPY

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

Molecular treatments in Duchenne muscular dystrophy

(2010) Michela Guglieri et al.   CURRENT OPINION IN PHARMACOLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Becker muscular dystrophy in Indian patients: Analysis of dystrophin gene deletion patterns

(2009) JayshreeJ Nadkarni et al.   NEUROLOGY INDIA

Human Splicing Finder: an online bioinformatics tool to predict splicing signals

(2009) François-Olivier Desmet et al.   NUCLEIC ACIDS RESEARCH

Integrated DNA, cDNA, and protein studies in Becker muscular dystrophy show high exception to the reading frame rule

(2008) Akanchha Kesari et al.   HUMAN MUTATION