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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

The New England journal of medicine, 2017-11, Vol.377 (18), p.1713-1722

2017

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INV04 Accounting for phenotypic variability in clinical outcomes

Neuromuscular disorders : NMD, 2023-10, Vol.33, p.S67-S67

Alfano, L.

2023

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A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design

Molecular genetics and metabolism, 2016-11, Vol.119 (3), p.239-248

2016

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NONO regulates the intra-S-phase checkpoint in response to UV radiation

Oncogene, 2016-02, Vol.35 (5), p.567-576

2016

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Cross-sectional data on the 100 meter timed test in boys with Duchenne muscular dystrophy compared to age-matched controls

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S186-S186

2016

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Establishing normative data for the 60 and 100-m run test in healthy male controls

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S240-S240

2015

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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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Evaluating the effect of a monetary incentive on performance of the 100-meter timed test in Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S186-S187

2016

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Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy

Journal of pharmacokinetics and pharmacodynamics, 2019-10, Vol.46 (5), p.441-455

2019

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Validity of ACTIVE for use across ambulatory abilities in Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S229-S230

2015

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Pilot data evaluating the utility of ACTIVE-mini as a biomarker in spinal muscular atrophy type 1

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S102-S102

2016

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S11 Long-term follow-up of the phase 1 START trial of onasemnogene abeparvovec gene therapy in spinal muscular atrophy type 1

Thorax, 2021-02, Vol.76 (Suppl 1), p.A10-A10

2021

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Comparison of ACTIVE-mini to the Vicon motion camera system in measuring infant movements

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S240-S241

2015

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A Novel Measure To Assess Self-Reported Physical Functioning In Patients With Sporadic Inclusion Body Myositis (Sibm)

Value in health, 2015-05, Vol.18 (3), p.A286-A286

2015

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Eteplirsen, a phosphorodiamidate morpholino oligomer (PMO) for Duchenne muscular dystrophy (DMD): Longitudinal comparison to external controls on six-minute walk test (6MWT) and loss of ambulation (LOA)

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S154-S154

2016

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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

The New England journal of medicine, 2017-11, Vol.377 (18), p.1713-1722

2017

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G.P.112

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

2014

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Eteplirsen, a Phosphorodiamidate morpholino oligomer (PMO) for the treatment of Duchenne muscular dystrophy (DMD): Clinical update

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S263-S263

2015

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Psychometric evaluation of the sporadic inclusion body myositis (sIBM) physical functioning assessment (sIFA)

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S239-S239

2015

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O13 Promoting an active lifestyle; use of an in-home body weight support system to increase exercise dosage for children with neuromuscular disease

Neuromuscular disorders : NMD, 2023-10, Vol.33, p.S132-S132

2023

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P321 Exploring hand and upper limb function in patients with inclusion body myositis

Neuromuscular disorders : NMD, 2023-10, Vol.33, p.S94-S94

2023

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P.066 AVXS-101 gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the phase 1 clinical trial

Canadian journal of neurological sciences, 2019-06, Vol.46 (s1), p.S32-S32

2019

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G.O.24

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.922-922

2014

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COVID-19 AND NEUROMUSCULAR DISEASES

Neuromuscular disorders : NMD, 2021-10, Vol.31, p.S56-S57

2021

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COVID-19 AND NEUROMUSCULAR DISEASES: EP.24 COVID-19 mitigation strategies for outcome measure assessments, training, and endpoint collection in clinic, clinical trials and natural history studies

Neuromuscular disorders : NMD, 2021-10, Vol.31, p.S56-S57

2021

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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

INV04 Accounting for phenotypic variability in clinical outcomes

A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design

NONO regulates the intra-S-phase checkpoint in response to UV radiation

Cross-sectional data on the 100 meter timed test in boys with Duchenne muscular dystrophy compared to age-matched controls

Establishing normative data for the 60 and 100-m run test in healthy male controls

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

Evaluating the effect of a monetary incentive on performance of the 100-meter timed test in Duchenne muscular dystrophy

Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy

Validity of ACTIVE for use across ambulatory abilities in Duchenne muscular dystrophy

Pilot data evaluating the utility of ACTIVE-mini as a biomarker in spinal muscular atrophy type 1

S11 Long-term follow-up of the phase 1 START trial of onasemnogene abeparvovec gene therapy in spinal muscular atrophy type 1

Comparison of ACTIVE-mini to the Vicon motion camera system in measuring infant movements

A Novel Measure To Assess Self-Reported Physical Functioning In Patients With Sporadic Inclusion Body Myositis (Sibm)

Eteplirsen, a phosphorodiamidate morpholino oligomer (PMO) for Duchenne muscular dystrophy (DMD): Longitudinal comparison to external controls on six-minute walk test (6MWT) and loss of ambulation (LOA)

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

G.P.112

Eteplirsen, a Phosphorodiamidate morpholino oligomer (PMO) for the treatment of Duchenne muscular dystrophy (DMD): Clinical update

Psychometric evaluation of the sporadic inclusion body myositis (sIBM) physical functioning assessment (sIFA)

O13 Promoting an active lifestyle; use of an in-home body weight support system to increase exercise dosage for children with neuromuscular disease

P321 Exploring hand and upper limb function in patients with inclusion body myositis

P.066 AVXS-101 gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the phase 1 clinical trial

G.O.24

COVID-19 AND NEUROMUSCULAR DISEASES

COVID-19 AND NEUROMUSCULAR DISEASES: EP.24 COVID-19 mitigation strategies for outcome measure assessments, training, and endpoint collection in clinic, clinical trials and natural history studies

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