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Synthesis of core/shell nanoparticles and their application in hydroxy-PAHs extraction

Journal of physics. Conference series, 2021-03, Vol.1853 (1), p.12020

2021

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

Annals of neurology, 2016-02, Vol.79 (2), p.257-271

2016

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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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G.O.25

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

2014

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Real-Time PCR for direct detection of Clostridium perfringens from horse with enterocolitis infection

Kufa Journal For Veterinary Medical Sciences, 2016-06, Vol.7 (1), p.74-80

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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The 100-meter timed test: ability to detect change over time in Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S235

2017

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P.464 - The 100-meter timed test: ability to detect change over time in Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S235-S235

2017

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DUCHENNE MUSCULAR DYSTROPHY - GENETICS

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S98-S99

2018

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DUCHENNE MUSCULAR DYSTROPHY - GENETICS: P.219Duchenne and Becker muscular dystrophy carriers: emerging evidence for a clinically important cardiomyopathy

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S98-S99

2018

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SMA – THERAPY

Neuromuscular disorders : NMD, 2020-10, Vol.30, p.S122-S123

2020

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DMD – THERAPY

Neuromuscular disorders : NMD, 2020-10, Vol.30, p.S128

2020

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DMD – THERAPY: P.280 Systemic gene transfer with rAAVrh74.MHCK7.micro-dystrophin in patients with Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2020-10, Vol.30, p.S128-S128

2020

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SMA – THERAPY: P.261 Long-term follow-up of onasemnogene abeparvovec gene therapy in spinal muscular atrophy type 1 (SMA1)

Neuromuscular disorders : NMD, 2020-10, Vol.30, p.S122-S123

2020

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Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): Long-term follow-up from the onasemnogene abeparvovec phase 1/2A clinical trial

Journal of the neurological sciences, 2019-10, Vol.405, p.271-271

2019

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DUCHENNE MUSCULAR DYSTROPHY - PHYSIOTHERAPY

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S126

2018

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DUCHENNE MUSCULAR DYSTROPHY - GENETICS: P.217Duchenne and Becker muscular dystrophy carrier mothers: characterization of skeletal and cardiac muscle compared to healthy controls

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S98-S98

2018

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DUCHENNE MUSCULAR DYSTROPHY - GENETICS

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S98

2018

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DUCHENNE MUSCULAR DYSTROPHY - PHYSIOTHERAPY

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S123

2018

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DUCHENNE MUSCULAR DYSTROPHY - PHYSIOTHERAPY: P.306Utility of the Bayley-III, North Star Ambulatory Assessment, and 100 meter timed test in quantifying gross motor delay in very young boys with Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S123-S123

2018

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SMA CLINICAL DATA, OUTCOME MEASURES AND REGISTRIES: P.89More than just fun and games: ACTIVE workspace volume video game quantifies meaningful change in function in individuals with spinal muscular atrophy

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S56-S57

2018

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SMA THERAPIES I

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S82

2018

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SMA CLINICAL DATA, OUTCOME MEASURES AND REGISTRIES

Neuromuscular disorders : NMD, 2018-10, Vol.28, p.S56-S57

2018

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Synthesis of core/shell nanoparticles and their application in hydroxy-PAHs extraction

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

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

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

G.O.25

Real-Time PCR for direct detection of Clostridium perfringens from horse with enterocolitis infection

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

The 100-meter timed test: ability to detect change over time in Duchenne muscular dystrophy

P.464 - The 100-meter timed test: ability to detect change over time in Duchenne muscular dystrophy

DUCHENNE MUSCULAR DYSTROPHY - GENETICS

DUCHENNE MUSCULAR DYSTROPHY - GENETICS: P.219Duchenne and Becker muscular dystrophy carriers: emerging evidence for a clinically important cardiomyopathy

SMA – THERAPY

DMD – THERAPY

DMD – THERAPY: P.280 Systemic gene transfer with rAAVrh74.MHCK7.micro-dystrophin in patients with Duchenne muscular dystrophy

SMA – THERAPY: P.261 Long-term follow-up of onasemnogene abeparvovec gene therapy in spinal muscular atrophy type 1 (SMA1)

Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): Long-term follow-up from the onasemnogene abeparvovec phase 1/2A clinical trial

DUCHENNE MUSCULAR DYSTROPHY - PHYSIOTHERAPY

DUCHENNE MUSCULAR DYSTROPHY - GENETICS: P.217Duchenne and Becker muscular dystrophy carrier mothers: characterization of skeletal and cardiac muscle compared to healthy controls

DUCHENNE MUSCULAR DYSTROPHY - GENETICS

DUCHENNE MUSCULAR DYSTROPHY - PHYSIOTHERAPY

DUCHENNE MUSCULAR DYSTROPHY - PHYSIOTHERAPY: P.306Utility of the Bayley-III, North Star Ambulatory Assessment, and 100 meter timed test in quantifying gross motor delay in very young boys with Duchenne muscular dystrophy

SMA CLINICAL DATA, OUTCOME MEASURES AND REGISTRIES: P.89More than just fun and games: ACTIVE workspace volume video game quantifies meaningful change in function in individuals with spinal muscular atrophy

SMA THERAPIES I

SMA CLINICAL DATA, OUTCOME MEASURES AND REGISTRIES

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