Cargando…
The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy
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 treatment. However, the low levels of the indu...
Autores principales: | Scaglioni, Dominic, Catapano, Francesco, Ellis, Matthew, Torelli, Silvia, Chambers, Darren, Feng, Lucy, Beck, Matthew, Sewry, Caroline, Monforte, Mauro, Harriman, Shawn, Koenig, Erica, Malhotra, Jyoti, Popplewell, Linda, Guglieri, Michela, Straub, Volker, Mercuri, Eugenio, Servais, Laurent, Phadke, Rahul, Morgan, Jennifer, Muntoni, Francesco |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2021
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7789286/ https://www.ncbi.nlm.nih.gov/pubmed/33407808 http://dx.doi.org/10.1186/s40478-020-01106-1 |
Ejemplares similares
-
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
por: Frank, Diane E., et al.
Publicado: (2020) -
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
por: Servais, Laurent, et al.
Publicado: (2022) -
A high–throughput digital script for multiplexed immunofluorescent analysis and quantification of sarcolemmal and sarcomeric proteins in muscular dystrophies
por: Scaglioni, Dominic, et al.
Publicado: (2020) -
Letter to the Editor: In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy
por: Muntoni, Francesco, et al.
Publicado: (2023) -
A novel high-throughput immunofluorescence analysis method for quantifying dystrophin intensity in entire transverse sections of Duchenne muscular dystrophy muscle biopsy samples
por: Sardone, Valentina, et al.
Publicado: (2018)