Cargando…

Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: Results of a double-blind randomized clinical trial

Duchenne muscular dystrophy (DMD) is a progressive, lethal neuromuscular disorder caused by the absence of dystrophin protein due to mutations of the dystrophin gene. Drisapersen is a 2′-O-methyl-phosphorothioate oligonucleotide designed to skip exon 51 in dystrophin pre-mRNA to restore the reading...

Descripción completa

Detalles Bibliográficos
Autores principales:	Flanigan, Kevin M., Voit, Thomas, Rosales, Xiomara Q., Servais, Laurent, Kraus, John E., Wardell, Claire, Morgan, Allison, Dorricott, Susie, Nakielny, Joanna, Quarcoo, Naashika, Liefaard, Lia, Drury, Tom, Campion, Giles, Wright, Padraig
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2013
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4145871/ https://www.ncbi.nlm.nih.gov/pubmed/24321374 http://dx.doi.org/10.1016/j.nmd.2013.09.004

Ejemplares similares

Placebo‐controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy
por: McDonald, Craig M., et al.
Publicado: (2018)

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study
por: Goemans, Nathalie M., et al.
Publicado: (2016)

A Movement Monitor Based on Magneto-Inertial Sensors for Non-Ambulant Patients with Duchenne Muscular Dystrophy: A Pilot Study in Controlled Environment
por: Le Moing, Anne-Gaëlle, et al.
Publicado: (2016)

Non-Ambulant Duchenne Patients Theoretically Treatable by Exon 53 Skipping have Severe Phenotype
por: Servais, Laurent, et al.
Publicado: (2015)

24 Month Longitudinal Data in Ambulant Boys with Duchenne Muscular Dystrophy
por: Mazzone, Elena Stacy, et al.
Publicado: (2013)

Preserved thenar muscles in non‐ambulant Duchenne muscular dystrophy patients
por: Naarding, Karin J., et al.
Publicado: (2021)

Peak functional ability and age at loss of ambulation in Duchenne muscular dystrophy
por: Zambon, Alberto A., et al.
Publicado: (2022)

Evaluation of Cardiac, Autonomic Functions in Ambulant Patients with Duchenne Muscular Dystrophy
por: Nayak, Amritharekha, et al.
Publicado: (2023)

Cholesterol metabolism is a potential therapeutic target in Duchenne muscular dystrophy
por: Amor, Fatima, et al.
Publicado: (2021)

Correction: 24 Month Longitudinal Data in Ambulant Boys with Duchenne Muscular Dystrophy
por: Mazzone, Elena Stacy, et al.
Publicado: (2013)

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
por: Mendell, Jerry R., et al.
Publicado: (2016)

Upper Limb Strength and Function Changes during a One-Year Follow-Up in Non-Ambulant Patients with Duchenne Muscular Dystrophy: An Observational Multicenter Trial
por: Seferian, Andreea Mihaela, et al.
Publicado: (2015)

MRI vastus lateralis fat fraction predicts loss of ambulation in Duchenne muscular dystrophy
por: Naarding, Karin J., et al.
Publicado: (2020)

The 6 Minute Walk Test and Performance of Upper Limb in Ambulant Duchenne Muscular Dystrophy Boys
por: Pane, Marika, et al.
Publicado: (2014)

Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients
por: McDonald, Craig M, et al.
Publicado: (2021)

miR-379 links glucocorticoid treatment with mitochondrial response in Duchenne muscular dystrophy
por: Sanson, M., et al.
Publicado: (2020)

Longitudinal Analysis of PUL 2.0 Domains in Ambulant and Non-Ambulant Duchenne Muscular Dystrophy Patients: How do they Change in Relation to Functional Ability?
por: Pane, Marika, et al.
Publicado: (2023)

Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes
por: Pane, Marika, et al.
Publicado: (2014)

Genotype characterization and delayed loss of ambulation by glucocorticoids in a large cohort of patients with Duchenne muscular dystrophy
por: Zhang, Shu, et al.
Publicado: (2021)

Lessons Learned from Discontinued Clinical Developments in Duchenne Muscular Dystrophy
por: Markati, Theodora, et al.
Publicado: (2021)

Correction: Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes
por: Pane, Marika, et al.
Publicado: (2015)

Correction: Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes
Publicado: (2015)

Evaluating the Feasibility and Reliability of Remotely Delivering and Scoring the North Star Ambulatory Assessment in Ambulant Patients with Duchenne Muscular Dystrophy
por: Emery, Nicholas, et al.
Publicado: (2022)

Early loss of ambulation is not a representative clinical feature in Duchenne muscular dystrophy dogs: remarks on the article of Barthélémy et al.
por: Duan, Dongsheng, et al.
Publicado: (2015)

RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect
por: Domenger, Claire, et al.
Publicado: (2017)

Genetic modifiers of ambulation in the cooperative international Neuromuscular research group Duchenne natural history study
por: Bello, Luca, et al.
Publicado: (2015)

Semi-Automated Analysis of Diaphragmatic Motion with Dynamic Magnetic Resonance Imaging in Healthy Controls and Non-Ambulant Subjects with Duchenne Muscular Dystrophy
por: Bishop, Courtney A., et al.
Publicado: (2018)

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)

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
por: Frank, Diane E., et al.
Publicado: (2020)

Newborn screening of duchenne muscular dystrophy specifically targeting deletions amenable to exon-skipping therapy
por: Beckers, Pablo, et al.
Publicado: (2021)

Benefits of glucocorticoids in non-ambulant boys/men with Duchenne muscular dystrophy: A multicentric longitudinal study using the Performance of Upper Limb test
por: Pane, Marika, et al.
Publicado: (2015)

Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53
por: Brogna, Claudia, et al.
Publicado: (2019)

A Sensitive, Reproducible and Objective Immunofluorescence Analysis Method of Dystrophin in Individual Fibers in Samples from Patients with Duchenne Muscular Dystrophy
por: Beekman, Chantal, et al.
Publicado: (2014)

Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy
por: Mariot, Virginie, et al.
Publicado: (2020)

Predictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophy
por: Barthélémy, Inès, et al.
Publicado: (2014)

Respiratory involvement in ambulant and non-ambulant patients with facioscapulohumeral muscular dystrophy
por: Moreira, Sandra, et al.
Publicado: (2017)

Correction: Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53
por: Brogna, Claudia, et al.
Publicado: (2019)

Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophy
por: Buyse, Gunnar M., et al.
Publicado: (2016)

Wearable full-body motion tracking of activities of daily living predicts disease trajectory in Duchenne muscular dystrophy
por: Ricotti, Valeria, et al.
Publicado: (2023)

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
por: Le Guiner, Caroline, et al.
Publicado: (2017)

Cannot write session to /tmp/vufind_sessions/sess_rofm52o3291pi7e8drp661gd5v