The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy
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The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy

Dominic Scaglioni, Francesco Catapano, Matthew Ellis, Silvia Torelli, Darren Chambers, Lucy Feng, Matthew Beck, Caroline Sewry, Mauro Monforte, Shawn Harriman, Erica Koenig, Jyoti Malhotra, Linda Popplewell, Michela Guglieri, Volker Straub, Eugenio Mercuri, Laurent Servais, Rahul Phadke, Jennifer Morgan, Francesco Muntoni;Dominic Scaglioni;Francesco Catapano;Matthew Ellis;Silvia Torelli;Darren Chambers;Lucy Feng;Matthew Beck;Caroline Sewry;Mauro Monforte;Shawn Harriman;Erica Koenig;Jyoti Malhotra;Linda Popplewell;Michela Guglieri;Volker Straub;Eugenio Mercuri;Laurent Servais;Rahul Phadke;Jennifer Morgan;Francesco Muntoni;
acta neuropathologica communications 2021 Vol. 9 pp. -
152
muntoni2021actathe

Abstract

During the last decade, multiple clinical trials for Duchenne muscular dystrophy (DMD) have focused on the induction of dystrophin expression using different strategies. Many of these trials have reported a clear increase in dystrophin protein following ...

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DOI:
10.1186/s40478-020-01106-1
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https://www.ncbi.nlm.nih.gov/pmc/articles/PMC77892...

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