Comprehensive review of adverse reactions and toxicology in ASO-based therapies for Duchenne Muscular Dystrophy: From FDA-approved drugs to peptide-conjugated ASO

Autor: Umme Sabrina Haque, Melissa Kohut, Toshifumi Yokota

Publikováno v: Current Research in Toxicology, Vol 7, Iss , Pp 100182- (2024)

Duchenne Muscular Dystrophy (DMD) is a devastating X-linked genetic disorder characterized by progressive muscle degeneration due to mutations in the dystrophin gene. This results in the absence or dysfunction of the dystrophin protein, leading to mu

Externí odkaz: https://doaj.org/article/4bc8b59de5f7483da8fd0386aaf8f26c

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

Autor: 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

Publikováno v: Acta Neuropathologica Communications, Vol 9, Iss 1, Pp 1-17 (2021)

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 prote

Externí odkaz: https://doaj.org/article/0bb99a8919a34521a7b32c5a0a245087

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Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial

Autor: Ashish Dugar, Eugenio Mercuri, Xiaodong Wang, Volker Straub, Baoguang Han, Daniela Leone, Navid Khan, Dan Wang, Erica Koenig, Laurent Servais, Andreea Mihaela Seferian, Michela Guglieri, Mariacristina Scoto, Francesco Muntoni

Publikováno v: Nucleic Acid Therapeutics
Nucleic Acid Therapeutics, In press, ⟨10.1089/nat.2021.0043⟩
Nucleic Acid Therapeutics, Mary Ann Liebert, Inc. publishers, In press, ⟨10.1089/nat.2021.0043⟩

The aim of this Phase 1/2, 2-part, multicenter trial was to report clinical safety and efficacy of long-term golodirsen treatment among ambulatory patients with exon 53 skip-amenable Duchenne muscular dystrophy (DMD). Part 1 was a 12-week, randomized

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::84e23f53949f6934fac11e79b70202fb
http://europepmc.org/articles/PMC8817703

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