Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

Exon skipping therapy for Duchenne muscular dystrophy

(2015) Ryszard Kole et al.   ADVANCED DRUG DELIVERY REVIEWS

AVI-7288 for Marburg Virus in Nonhuman Primates and Humans

(2015) Alison E. Heald et al.   NEW ENGLAND JOURNAL OF MEDICINE

Characterization of pulmonary function in Duchenne Muscular Dystrophy

(2015) O.H. Mayer et al.   PEDIATRIC PULMONOLOGY

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

(2014) Thomas Voit et al.   LANCET NEUROLOGY

Six-Minute Walk Test: Reference Values and Prediction Equation in Healthy Boys Aged 5 to12 Years

(2014) Nathalie Goemans et al.   PLoS One

6 Minute Walk Test in Duchenne MD Patients with Different Mutations: 12 Month Changes

(2014) Marika Pane et al.   PLoS One

Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes

(2014) Marika Pane et al.   PLoS One

Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

The cooperative international neuromuscular research group duchenne natural history study-a longitudinal investigation in the era of glucocorticoid therapy: Design of protocol and the methods used

(2013) Craig M. McDonald et al.   MUSCLE & NERVE

THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study

(2013) Craig M. Mcdonald et al.   MUSCLE & NERVE

The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and othe

(2013) Erik K. Henricson et al.   MUSCLE & NERVE

The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study

(2013) Craig M. McDonald et al.   MUSCLE & NERVE

T.I.2 Exon skipping and PRO044 in Duchenne muscular dystrophy: Extending the program

(2013) A. Ferlini et al.   NEUROMUSCULAR DISORDERS

Ambulatory capacity and disease progression as measured by the 6-minute-walk-distance in Duchenne muscular dystrophy subjects on daily corticosteroids

(2013) Nathalie Goemans et al.   NEUROMUSCULAR DISORDERS

Respiratory muscle decline in duchenne muscular dystrophy

(2013) Sonia Khirani et al.   PEDIATRIC PULMONOLOGY

24 Month Longitudinal Data in Ambulant Boys with Duchenne Muscular Dystrophy

(2013) Elena Stacy Mazzone et al.   PLoS One

Chemical and Mechanistic Toxicology Evaluation of Exon Skipping Phosphorodiamidate Morpholino Oligomers in mdx Mice

(2011) Peter Sazani et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

Repeat-Dose Toxicology Evaluation in Cynomolgus Monkeys of AVI-4658, a Phosphorodiamidate Morpholino Oligomer (PMO) Drug for the Treatment of Duchenne Muscular Dystrophy

(2011) Peter Sazani et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

Restoration of the Dystrophin-associated Glycoprotein Complex After Exon Skipping Therapy in Duchenne Muscular Dystrophy

(2011) Sebahattin Cirak et al.   MOLECULAR THERAPY

Targeting RNA to treat neuromuscular disease

(2011) Francesco Muntoni et al.   NATURE REVIEWS DRUG DISCOVERY

Functional changes in Duchenne muscular dystrophy: A 12-month longitudinal cohort study

(2011) E. Mazzone et al.   NEUROLOGY

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

Safety Pharmacology and Genotoxicity Evaluation of AVI-4658

(2010) Peter Sazani et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

The 6-minute walk test in Duchenne/Becker muscular dystrophy: Longitudinal observations

(2010) Craig M. MCDonald et al.   MUSCLE & NERVE

North Star Ambulatory Assessment, 6-minute walk test and timed items in ambulant boys with Duchenne muscular dystrophy

(2010) Elena Mazzone et al.   NEUROMUSCULAR DISORDERS

Delayed Diagnosis in Duchenne Muscular Dystrophy: Data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet)

(2009) Emma Ciafaloni et al.   JOURNAL OF PEDIATRICS

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

(2009) Maria Kinali et al.   LANCET NEUROLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy

(2009) Craig M. McDonald et al.   MUSCLE & NERVE

149th ENMC International Workshop and 1st TREAT-NMD Workshop on: “Planning Phase I/II Clinical trials using Systemically Delivered Antisense Oligonucleotides in Duchenne Muscular Dystrophy”

(2008) Francesco Muntoni et al.   NEUROMUSCULAR DISORDERS