The myotonic dystrophies: molecular, clinical, and therapeutic challenges

B Udd, R Krahe - The Lancet. Neurology, 2012 - pubmed.ncbi.nlm.nih.gov
Myotonic dystrophy is the most common type of muscular dystrophy in adults and is
characterised by progressive myopathy, myotonia, and multiorgan involvement. Two …

[引用][C] The myotonic dystrophies: molecular, clinical, and therapeutic challenges

B Udd, R Krahe - The Lancet Neurology, 2012 - Elsevier Limited
[PDF] researchgate.net

[PDF][PDF] The myotonic dystrophies: molecular, clinical, and therapeutic challenges

B Udd, R Krahe - Lancet Neurol, 2012 - researchgate.net
RNA-binding proteins, resulting in a spliceopathy of downstream effector genes. The
prevailing paradigm therefore is that both disorders are toxic RNA diseases. However …