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Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

Lancet neurology, 2021-12, Vol.20 (12), p.1012-1026

2021

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Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial

Lancet neurology, 2021-12, Vol.20 (12), p.1027-1037

2021

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Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial

JAMA neurology, 2023-06, Vol.80 (6), p.558

2023

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104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07)

Journal of neurology, 2024-05, Vol.271 (5), p.2810-2823

2024

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Motor neuron involvement expands the neuropathological phenotype of late‐onset ataxia in RFC1 mutation (CANVAS)

Brain pathology (Zurich, Switzerland), 2022-07, Vol.32 (4), p.e13051-n/a

2022

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High prevalence of paraspinal muscle involvement in adults with McArdle disease

Muscle & nerve, 2022-05, Vol.65 (5), p.568-573

2022

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Drug‐refractory myasthenia gravis: Clinical characteristics, treatments, and outcome

Annals of clinical and translational neurology, 2022-02, Vol.9 (2), p.122-131

2022

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Equivalente migrañoso de inicio tardío con aura prolongada y alteración de la perfusión cerebral

Revista de neurologiá, 2020-06, Vol.70 (11), p.430

2020

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Pathophysiological Underpinnings of Extra-Motor Neurodegeneration in Amyotrophic Lateral Sclerosis: New Insights From Biomarker Studies

Frontiers in neurology, 2022-01, Vol.12, p.750543-750543

2022

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Correlation Between Respiratory Accessory Muscles and Diaphragm Pillars MRI and Pulmonary Function Test in Late-Onset Pompe Disease Patients

Frontiers in neurology, 2021-03, Vol.12, p.621257-621257

2021

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Magnetization Transfer Ratio in Lower Limbs of Late Onset Pompe Patients Correlates With Intramuscular Fat Fraction and Muscle Function Tests

Frontiers in neurology, 2021-03, Vol.12, p.634766-634766

2021

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Corrigendum: Magnetization Transfer Ratio in Lower Limbs of Late Onset Pompe Patients Correlates With Intramuscular Fat Fraction and Muscle Function Tests

Frontiers in neurology, 2021-07, Vol.12, p.727020-727020

2021

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Late-onset migraine equivalent with prolonged aura and altered brain perfusion

Revista de neurologiá, 2020-06, Vol.70 (11), p.430

2020

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Azathioprine therapy induces selective NK cell depletion and IFN-γ deficiency predisposing to herpesvirus reactivation

Journal of allergy and clinical immunology, 2023-01, Vol.151 (1), p.280-286.e2

2023

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Rituximab in myasthenia gravis: efficacy, associated infections and risk of induced hypogammaglobulinemia

Neuromuscular disorders : NMD, 2022-08, Vol.32 (8), p.664-671

2022

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Inter-laboratory comparison of routine autoantibody detection methods for autoimmune neuropathies and myasthenia gravis

Journal of neurology, 2024-07, Vol.271 (7), p.4119-4130

2024

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Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial

Lancet neurology, 2024-05, Vol.23 (5), p.477-486

2024

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Phenotype and clinical outcomes of Glu89Lys hereditary transthyretin amyloidosis: a new endemic variant in Spain

Amyloid, 2023-04, Vol.30 (2), p.199-207

2023

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Psychotic symptoms in drug resistant epilepsy patients after cortical stimulation

Epilepsy research, 2021-07, Vol.173, p.106630-106630, Article 106630

2021

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Description of clinical and genetic features of 122 patients included in the Spanish Pompe registry

Neuromuscular disorders : NMD, 2024-01, Vol.34, p.1-8

2024

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Immune Response and Safety of SARS-CoV-2 mRNA-1273 Vaccine in Patients With Myasthenia Gravis

Neurology : neuroimmunology & neuroinflammation, 2022-07, Vol.9 (4)

2022

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Reduced Number of Thymoma CTLA4-Positive Cells Is Associated With a Higher Probability of Developing Myasthenia Gravis

Neurology : neuroimmunology & neuroinflammation, 2023-03, Vol.10 (2)

2023

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Neuroinflammation-Related Proteins NOD2 and Spp1 Are Abnormally Upregulated in Amyotrophic Lateral Sclerosis

Neurology : neuroimmunology & neuroinflammation, 2023-03, Vol.10 (2)

2023

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A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week Study of the Efficacy and Safety of Losmapimod in Subjects with FSHD: ReDUX4 (S23.007)

Neurology, 2022-05, Vol.98 (18_supplement)

2022

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Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial

Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial

104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07)

Motor neuron involvement expands the neuropathological phenotype of late‐onset ataxia in RFC1 mutation (CANVAS)

High prevalence of paraspinal muscle involvement in adults with McArdle disease

Drug‐refractory myasthenia gravis: Clinical characteristics, treatments, and outcome

Equivalente migrañoso de inicio tardío con aura prolongada y alteración de la perfusión cerebral

Pathophysiological Underpinnings of Extra-Motor Neurodegeneration in Amyotrophic Lateral Sclerosis: New Insights From Biomarker Studies

Correlation Between Respiratory Accessory Muscles and Diaphragm Pillars MRI and Pulmonary Function Test in Late-Onset Pompe Disease Patients

Magnetization Transfer Ratio in Lower Limbs of Late Onset Pompe Patients Correlates With Intramuscular Fat Fraction and Muscle Function Tests

Corrigendum: Magnetization Transfer Ratio in Lower Limbs of Late Onset Pompe Patients Correlates With Intramuscular Fat Fraction and Muscle Function Tests

Late-onset migraine equivalent with prolonged aura and altered brain perfusion

Azathioprine therapy induces selective NK cell depletion and IFN-γ deficiency predisposing to herpesvirus reactivation

Rituximab in myasthenia gravis: efficacy, associated infections and risk of induced hypogammaglobulinemia

Inter-laboratory comparison of routine autoantibody detection methods for autoimmune neuropathies and myasthenia gravis

Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial

Phenotype and clinical outcomes of Glu89Lys hereditary transthyretin amyloidosis: a new endemic variant in Spain

Psychotic symptoms in drug resistant epilepsy patients after cortical stimulation

Description of clinical and genetic features of 122 patients included in the Spanish Pompe registry

Immune Response and Safety of SARS-CoV-2 mRNA-1273 Vaccine in Patients With Myasthenia Gravis

Reduced Number of Thymoma CTLA4-Positive Cells Is Associated With a Higher Probability of Developing Myasthenia Gravis

Neuroinflammation-Related Proteins NOD2 and Spp1 Are Abnormally Upregulated in Amyotrophic Lateral Sclerosis

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week Study of the Efficacy and Safety of Losmapimod in Subjects with FSHD: ReDUX4 (S23.007)

Reachable Workspace to Evaluate Efficacy of Losmapimod in Subjects with FSHD in Two Phase 2 Studies (P4-13.008)

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