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Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps

Developmental medicine and child neurology, 2019-01, Vol.61 (1), p.19-24

2019

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Normalized grip strength is a sensitive outcome measure through all stages of Duchenne muscular dystrophy

Journal of neurology, 2020-07, Vol.267 (7), p.2022-2028

2020

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Natural history of Type 2 and 3 spinal muscular atrophy: 2‐year NatHis‐SMA study

Annals of clinical and translational neurology, 2021-02, Vol.8 (2), p.359-373

2021

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Quantitative nuclear magnetic resonance imaging detects subclinical changes over 1 year in skeletal muscle of GNE myopathy

Journal of neurology, 2020, Vol.267 (1), p.228-238

2020

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Nuclear poly(A)-binding protein aggregates misplace a pre-mRNA outside of SC35 speckle causing its abnormal splicing

Nucleic acids research, 2016-12, Vol.44 (22), p.10929-10945

2016

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A negative feedback loop between fibroadipogenic progenitors and muscle fibres involving endothelin promotes human muscle fibrosis

Journal of cachexia, sarcopenia and muscle, 2022-06, Vol.13 (3), p.1771-1784

2022

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Mitochondrial dysfunction reveals the role of mRNA poly(A) tail regulation in oculopharyngeal muscular dystrophy pathogenesis

PLoS genetics, 2015-03, Vol.11 (3), p.e1005092-e1005092

2015

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Invited review: Stem cells and muscle diseases: advances in cell therapy strategies

Neuropathology and applied neurobiology, 2015-04, Vol.41 (3), p.270-287

2015

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Hereditary inclusion-body myopathy: Clues on pathogenesis and possible therapy

Muscle & nerve, 2009-09, Vol.40 (3), p.340-349

2009

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Hyposialylation of neprilysin possibly affects its expression and enzymatic activity in hereditary inclusion-body myopathy muscle

Journal of neurochemistry, 2008-05, Vol.105 (3), p.971-981

2008

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Upper limb disease evolution in exon 53 skipping eligible patients with Duchenne muscular dystrophy

Annals of clinical and translational neurology, 2021-10, Vol.8 (10), p.1938-1950

2021

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Increased aging in primary muscle cultures of sporadic inclusion-body myositis

Neurobiology of aging, 2010-07, Vol.31 (7), p.1205-1214

2010

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Leveraging Natural History Data in One- and Two-Arm Hierarchical Bayesian Studies of Rare Disease Progression

Statistics in biosciences, 2022-07, Vol.14 (2), p.237-258

2022

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MyoD Expression Restores Defective Myogenic Differentiation of Human Mesoangioblasts from Inclusion-Body Myositis Muscle

Proceedings of the National Academy of Sciences - PNAS, 2006-11, Vol.103 (45), p.16995-17000

2006

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Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy

Neurology, 2020-05, Vol.94 (21), p.e2270

2020

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Mesoangioblasts from Facioscapulohumeral Muscular Dystrophy Display in Vivo a Variable Myogenic Ability Predictable by their in Vitro Behavior

Cell transplantation, 2011-01, Vol.20 (8), p.1299-1313

2011

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Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

The New England journal of medicine, 2018-02, Vol.378 (7), p.625-635

2018

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Nusinersen in patients older than 7 months with spinal muscular atrophy type 1: A cohort study

Neurology, 2018-10, Vol.91 (14), p.e1312

2018

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Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

Lancet neurology, 2022-01, Vol.21 (1), p.42-52

2022

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X-linked myotubular myopathy: A prospective international natural history study

Neurology, 2019-04, Vol.92 (16), p.e1852

2019

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JEWELFISH: 24-month Safety, Pharmacodynamic and Exploratory Efficacy Data in Non-Treatment-Naïve Patients with Spinal Muscular Atrophy (SMA) Receiving Treatment with Risdiplam (P7-9.004)

Neurology, 2023-04, Vol.100 (17_supplement_2)

2023

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Home-Based Monitor for Gait and Activity Analysis

Journal of visualized experiments, 2019-08 (150)

2019

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Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps

Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study

Normalized grip strength is a sensitive outcome measure through all stages of Duchenne muscular dystrophy

Natural history of Type 2 and 3 spinal muscular atrophy: 2‐year NatHis‐SMA study

Upper limb strength and function changes during a one-year follow-up in non-ambulant patients with Duchenne Muscular Dystrophy: an observational multicenter trial

Quantitative nuclear magnetic resonance imaging detects subclinical changes over 1 year in skeletal muscle of GNE myopathy

Nuclear poly(A)-binding protein aggregates misplace a pre-mRNA outside of SC35 speckle causing its abnormal splicing

A negative feedback loop between fibroadipogenic progenitors and muscle fibres involving endothelin promotes human muscle fibrosis

Mitochondrial dysfunction reveals the role of mRNA poly(A) tail regulation in oculopharyngeal muscular dystrophy pathogenesis

Upper limb evaluation and one-year follow up of non-ambulant patients with spinal muscular atrophy: an observational multicenter trial

Invited review: Stem cells and muscle diseases: advances in cell therapy strategies

Hereditary inclusion-body myopathy: Clues on pathogenesis and possible therapy

Hyposialylation of neprilysin possibly affects its expression and enzymatic activity in hereditary inclusion-body myopathy muscle

Upper limb disease evolution in exon 53 skipping eligible patients with Duchenne muscular dystrophy

Increased aging in primary muscle cultures of sporadic inclusion-body myositis

Leveraging Natural History Data in One- and Two-Arm Hierarchical Bayesian Studies of Rare Disease Progression

MyoD Expression Restores Defective Myogenic Differentiation of Human Mesoangioblasts from Inclusion-Body Myositis Muscle

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy

Mesoangioblasts from Facioscapulohumeral Muscular Dystrophy Display in Vivo a Variable Myogenic Ability Predictable by their in Vitro Behavior

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

Nusinersen in patients older than 7 months with spinal muscular atrophy type 1: A cohort study

Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

X-linked myotubular myopathy: A prospective international natural history study

JEWELFISH: 24-month Safety, Pharmacodynamic and Exploratory Efficacy Data in Non-Treatment-Naïve Patients with Spinal Muscular Atrophy (SMA) Receiving Treatment with Risdiplam (P7-9.004)

Home-Based Monitor for Gait and Activity Analysis

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