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A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease

The New England journal of medicine, 2010-04, Vol.362 (15), p.1396-1406

2010

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Clinical features of late-onset Pompe disease: A prospective cohort study

Muscle & nerve, 2008-10, Vol.38 (4), p.1236-1245

2008

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Cobomarsen, an Oligonucleotide Inhibitor of miR-155, Slows DLBCL Tumor Cell Growth In Vitro and In Vivo

Clinical cancer research, 2021-02, Vol.27 (4), p.1139-1149

2021

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CINRG pilot trial of coenzyme Q10 in steroid-treated duchenne muscular dystrophy

Muscle & nerve, 2011-08, Vol.44 (2), p.174-178

2011

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Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations

Annals of neurology, 2001-06, Vol.49 (6), p.706-711

2001

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A Phase I/II trial of MYO-029 in Adult Subjects with Muscular Dystrophy

Annals of neurology, 2008-05, Vol.63 (5), p.561-571

2008

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Barriers and facilitators to clinical trial participation among parents of children with pediatric neuromuscular disorders

Clinical trials (London, England), 2018-04, Vol.15 (2), p.139-148

2018

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Challenges in drug development for muscle disease: A stakeholders' meeting

Muscle & Nerve, 2007-01, Vol.35 (1), p.8-16

2007

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Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy

Muscle & nerve, 2007-01, Vol.35 (1), p.36-42

2007

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CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy

Annals of neurology, 2005-07, Vol.58 (1), p.151-155

2005

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Congenital muscular dystrophies

Handbook of Clinical Neurology, 2011, Vol.101, p.47-79

2011

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Liquid formulation of pentoxifylline is a poorly tolerated treatment for duchenne dystrophy

Muscle & nerve, 2011-08, Vol.44 (2), p.170-173

2011

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CINRG pilot trial of oxatomide in steroid-naïve Duchenne muscular dystrophy

European journal of paediatric neurology, 2007-11, Vol.11 (6), p.337-340

2007

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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 other commonly used clinical trial outcome measures

Muscle & nerve, 2013-07, Vol.48 (1), p.55-67

2013

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Worsening of multifocal motor neuropathy during pregnancy

Neurology, 2002-07, Vol.59 (1), p.139-141

2002

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A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy

Annals of neurology, 2008-05, Vol.63 (5), p.561-571

2008

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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

Muscle & nerve, 2013-07, Vol.48 (1), p.32-54

2013

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Genome-wide homozygosity mapping localizes a gene for autosomal recessive non-progressive infantile ataxia to 20q11-q13

Human genetics, 2003-08, Vol.113 (3), p.293-295

2003

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Severe autosomal recessive rippling muscle disease

Muscle & nerve, 2001-11, Vol.24 (11), p.1542-1547

2001

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Collaborative translational research leading to multicenter clinical trials in Duchenne muscular dystrophy: the Cooperative International Neuromuscular Research Group (CINRG)

Neuromuscular disorders : NMD, 2002-10, Vol.12, p.S147-S154

2002

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Update of the Pompe disease mutation database with 107 sequence variants and a format for severity rating

Human mutation, 2008-06, Vol.29 (6), p.E13-E26

2008

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Pharmacologic and genetic therapy for childhood muscular dystrophies

Current neurology and neuroscience reports, 2001-03, Vol.1 (2), p.168-174

2001

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Cardiovascular abnormalities in late-onset Pompe disease and response to enzyme replacement therapy

Genetics in medicine, 2011-07, Vol.13 (7), p.625-631

2011

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A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease

Clinical features of late-onset Pompe disease: A prospective cohort study

Cobomarsen, an Oligonucleotide Inhibitor of miR-155, Slows DLBCL Tumor Cell Growth In Vitro and In Vivo

CINRG pilot trial of coenzyme Q10 in steroid-treated duchenne muscular dystrophy

Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations

Functional and molecular effects of arginine butyrate and prednisone on muscle and heart in the mdx mouse model of Duchenne Muscular Dystrophy

A Phase I/II trial of MYO-029 in Adult Subjects with Muscular Dystrophy

Barriers and facilitators to clinical trial participation among parents of children with pediatric neuromuscular disorders

Challenges in drug development for muscle disease: A stakeholders' meeting

Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy

CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy

Congenital muscular dystrophies

Liquid formulation of pentoxifylline is a poorly tolerated treatment for duchenne dystrophy

CINRG pilot trial of oxatomide in steroid-naïve Duchenne muscular dystrophy

Worsening of multifocal motor neuropathy during pregnancy

A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy

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

Genome-wide homozygosity mapping localizes a gene for autosomal recessive non-progressive infantile ataxia to 20q11-q13

Severe autosomal recessive rippling muscle disease

Collaborative translational research leading to multicenter clinical trials in Duchenne muscular dystrophy: the Cooperative International Neuromuscular Research Group (CINRG)

Update of the Pompe disease mutation database with 107 sequence variants and a format for severity rating

Pharmacologic and genetic therapy for childhood muscular dystrophies

Cardiovascular abnormalities in late-onset Pompe disease and response to enzyme replacement therapy

Correction: Functional and Molecular Effects of Arginine Butyrate and Prednisone on Muscle and Heart in the mdx Mouse Model of Duchenne Muscular Dystrophy

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