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O13 Progress toward molecular-based therapies for muscular dystrophy

Neuromuscular disorders : NMD, 2014-03, Vol.24, p.S4-S4

Mendell, J.R

2014

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T.I.1 Potential for gene therapy in DMD

Neuromuscular disorders : NMD, 2011-10, Vol.21 (9), p.701-702

Mendell, J.R

2011

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The 2-min walk test is sufficient for evaluating walking abilities in sporadic inclusion body myositis

Neuromuscular disorders : NMD, 2014-03, Vol.24 (3), p.222-226

2014

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Randomized controlled trial of IVIg in untreated chronic inflammatory demyelinating polyradiculoneuropathy

Neurology, 2001-02, Vol.56 (4), p.445-449

2001

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Long-term treatment with the phosphorodiamidate morpholino oligomer eteplirsen in patients with Duchenne muscular dystrophy amenable to exon 51 skipping: analysis of dystrophin production

Neuromuscular disorders : NMD, 2017-03, Vol.27, p.S16-S16

2017

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Development of a validated Western blot method for quantification of human dystrophin protein used in phase 2 and 3 clinical trials of eteplirsen for the treatment of Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2017-03, Vol.27, p.S16-S16

2017

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Is cardiac dysfunction a feature of dysferlinopathy? Data from the Clinical Outcome Study of Dysferlinopathy

Neuromuscular disorders : NMD, 2018-04, Vol.28, p.S5-S6

2018

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Identification of proximal spinal muscular atrophy carriers and patients by analysis of SMNT and SMNC gene copy number

American journal of human genetics, 1997-06, Vol.60 (6), p.1411-1422

1997

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G.O.9

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.851-852

2014

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G.P.112

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.828-829

2014

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G.O.26

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.923-923

2014

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G.P.227

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.885-885

2014

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T.P.38

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.905-905

2014

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P.2.10 A shorter timed walking or running test may be sufficient for testing function in Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.751-752

2013

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Superficial peroneal nerve/peroneus brevis muscle biopsy in vasculitic neuropathy

Neurology, 2000-09, Vol.55 (5), p.636-643

2000

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P.14.17 Evaluation of the ability of timed walking tests to quantify function in sporadic inclusion body myositis

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.818-818

2013

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P.6.2 Development of a proof-of-concept device using the Microsoft Kinect to assess movement in infants with spinal muscular atrophy

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.770-770

2013

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P.14.16 Longitudinal changes in strength and functional outcomes in sporadic inclusion body myositis

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.818-818

2013

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G.P.289

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.904-904

2014

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T.P.1

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.860-860

2014

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Intragenic telSMN Mutations: Frequency, Distribution, Evidence of a Founder Effect, and Modification of the Spinal Muscular Atrophy Phenotype by cenSMN Copy Number

American journal of human genetics, 1998-12, Vol.63 (6), p.1712-1723

1998

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G.O.16

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.919-920

2014

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S.P.3 Diverse walking distances predict functional outcomes in Duchenne Muscular Dystrophy

Neuromuscular disorders : NMD, 2012-10, Vol.22 (9), p.876-876

2012

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S.P.11 Development of a virtual upper extremity assessment tool for individuals with DMD across the lifespan

Neuromuscular disorders : NMD, 2012-10, Vol.22 (9), p.878-879

2012

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G.P.167

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.855-856

2014

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O13 Progress toward molecular-based therapies for muscular dystrophy

T.I.1 Potential for gene therapy in DMD

The 2-min walk test is sufficient for evaluating walking abilities in sporadic inclusion body myositis

Randomized controlled trial of IVIg in untreated chronic inflammatory demyelinating polyradiculoneuropathy

Long-term treatment with the phosphorodiamidate morpholino oligomer eteplirsen in patients with Duchenne muscular dystrophy amenable to exon 51 skipping: analysis of dystrophin production

Development of a validated Western blot method for quantification of human dystrophin protein used in phase 2 and 3 clinical trials of eteplirsen for the treatment of Duchenne muscular dystrophy

Is cardiac dysfunction a feature of dysferlinopathy? Data from the Clinical Outcome Study of Dysferlinopathy

Identification of proximal spinal muscular atrophy carriers and patients by analysis of SMNT and SMNC gene copy number

G.O.9

G.P.112

G.O.26

G.P.227

T.P.38

P.2.10 A shorter timed walking or running test may be sufficient for testing function in Duchenne muscular dystrophy

Superficial peroneal nerve/peroneus brevis muscle biopsy in vasculitic neuropathy

P.14.17 Evaluation of the ability of timed walking tests to quantify function in sporadic inclusion body myositis

P.6.2 Development of a proof-of-concept device using the Microsoft Kinect to assess movement in infants with spinal muscular atrophy

P.14.16 Longitudinal changes in strength and functional outcomes in sporadic inclusion body myositis

G.P.289

T.P.1

Intragenic telSMN Mutations: Frequency, Distribution, Evidence of a Founder Effect, and Modification of the Spinal Muscular Atrophy Phenotype by cenSMN Copy Number

G.O.16

S.P.3 Diverse walking distances predict functional outcomes in Duchenne Muscular Dystrophy

S.P.11 Development of a virtual upper extremity assessment tool for individuals with DMD across the lifespan

G.P.167

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