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Pascal Laforêt

Showing results (101-110 of 164) with videos related to

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Journal of Neurology|January 3, 2021
Development of new outcome measures for adult SMA type III and IV: a multimodal longitudinal studyGiorgia Querin, Timothée Lenglet, Rabab Debs, et al.
The Lancet. Neurology|November 20, 2021
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 trialBenedikt Schoser, Mark Roberts, Barry J Byrne, et al.
Neurology|October 18, 2019
Large variation in effects during 10 years of enzyme therapy in adults with Pompe diseaseLaurike Harlaar, Jean-Yves Hogrel, Barbara Perniconi, et al.
The Lancet. Neurology|February 22, 2025
Clinical insights in enzyme replacement therapy for metabolic storage disorders: lessons from Pompe diseaseNadine A M E van der Beek, Maudy T M Theunissen, Johanna M P van den Hout, et al.
Molecular Genetics and Metabolism|October 4, 2012
Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfaAns T van der Ploeg, Richard Barohn, Lisa Carlson, et al.
European Journal of Neurology|May 11, 2023
Caveolinopathy: Clinical, histological, and muscle imaging features and follow-up in a multicenter retrospective cohortEdouard Berling, Camille Verebi, Nadia Venturelli, et al.
Neurology|October 25, 2013
Episodic weakness due to mitochondrial DNA MT-ATP6/8 mutationsKarine Auré, Odile Dubourg, Claude Jardel, et al.
Scientific Reports|November 5, 2016
Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patientsElisa Masat, Pascal Laforêt, Marie De Antonio, et al.
Journal of Inherited Metabolic Disease|June 10, 2020
Long-term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe RegistryClaudio Semplicini, Marie De Antonio, Nadjib Taouagh, et al.
Annals of Clinical and Translational Neurology|September 15, 2019
No effect of triheptanoin on exercise performance in McArdle diseaseKaren L Madsen, Pascal Laforêt, Astrid E Buch, et al.
Pageof 17

Showing results (101-110 of 164) with videos related to

Sort By:
Pageof 17
Journal of Neurology|January 3, 2021
Development of new outcome measures for adult SMA type III and IV: a multimodal longitudinal studyGiorgia Querin, Timothée Lenglet, Rabab Debs, et al.
The Lancet. Neurology|November 20, 2021
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 trialBenedikt Schoser, Mark Roberts, Barry J Byrne, et al.
Neurology|October 18, 2019
Large variation in effects during 10 years of enzyme therapy in adults with Pompe diseaseLaurike Harlaar, Jean-Yves Hogrel, Barbara Perniconi, et al.
The Lancet. Neurology|February 22, 2025
Clinical insights in enzyme replacement therapy for metabolic storage disorders: lessons from Pompe diseaseNadine A M E van der Beek, Maudy T M Theunissen, Johanna M P van den Hout, et al.
Molecular Genetics and Metabolism|October 4, 2012
Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfaAns T van der Ploeg, Richard Barohn, Lisa Carlson, et al.
European Journal of Neurology|May 11, 2023
Caveolinopathy: Clinical, histological, and muscle imaging features and follow-up in a multicenter retrospective cohortEdouard Berling, Camille Verebi, Nadia Venturelli, et al.
Neurology|October 25, 2013
Episodic weakness due to mitochondrial DNA MT-ATP6/8 mutationsKarine Auré, Odile Dubourg, Claude Jardel, et al.
Scientific Reports|November 5, 2016
Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patientsElisa Masat, Pascal Laforêt, Marie De Antonio, et al.
Journal of Inherited Metabolic Disease|June 10, 2020
Long-term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe RegistryClaudio Semplicini, Marie De Antonio, Nadjib Taouagh, et al.
Annals of Clinical and Translational Neurology|September 15, 2019
No effect of triheptanoin on exercise performance in McArdle diseaseKaren L Madsen, Pascal Laforêt, Astrid E Buch, et al.
Pageof 17