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Astrid Pechmann

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Journal of Neuromuscular Diseases|November 21, 2019
Treatment with Nusinersen - Challenges Regarding the Indication for Children with SMA Type 1Astrid Pechmann, Matthias Baumann, Günther Bernert, et al.
The Lancet. Neurology|March 22, 2020
Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort studyTim Hagenacker, Claudia D Wurster, René Günther, et al.
The Lancet Regional Health. Europe|October 22, 2024
Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational studyClaudia Weiß, Lena-Luise Becker, Johannes Friese, et al.
Expert Review of Neurotherapeutics|November 13, 2025
A plain language summary of the SAPPHIRE clinical trial of apitegromab in children and young adults with spinal muscular atrophyThomas O Crawford, Laurent Servais, Eugenio Mercuri, et al.
The Lancet. Neurology|August 16, 2025
Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trialThomas O Crawford, Laurent Servais, Eugenio Mercuri, et al.
The Lancet. Child & Adolescent Health|November 10, 2021
Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort studyClaudia Weiß, Andreas Ziegler, Lena-Luise Becker, et al.
Journal of Neuromuscular Diseases|December 4, 2022
Newbornscreening SMA - From Pilot Project to Nationwide Screening in GermanyWolfgang Müller-Felber, Astrid Blaschek, Oliver Schwartz, et al.
Journal of Neuromuscular Diseases|September 12, 2025
Phenotypic intrafamilial variability of 5q-associated spinal muscular atrophy: A systematic multicentre sibling studyBenedikt Becker, Isabell Cordts, Jutta Becker, et al.
Brain : a Journal of Neurology|July 20, 2022
Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophyAstrid Pechmann, Max Behrens, Katharina Dörnbrack, et al.
Journal of Neuromuscular Diseases|November 24, 2025
Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland-part I-systematic literature review and existing evidenceClaudia Weiß, Katharina Vill, Matthias Baumann, et al.
Pageof 6

Showing results (41-50 of 55) with videos related to

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Pageof 6
Journal of Neuromuscular Diseases|November 21, 2019
Treatment with Nusinersen - Challenges Regarding the Indication for Children with SMA Type 1Astrid Pechmann, Matthias Baumann, Günther Bernert, et al.
The Lancet. Neurology|March 22, 2020
Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort studyTim Hagenacker, Claudia D Wurster, René Günther, et al.
The Lancet Regional Health. Europe|October 22, 2024
Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational studyClaudia Weiß, Lena-Luise Becker, Johannes Friese, et al.
Expert Review of Neurotherapeutics|November 13, 2025
A plain language summary of the SAPPHIRE clinical trial of apitegromab in children and young adults with spinal muscular atrophyThomas O Crawford, Laurent Servais, Eugenio Mercuri, et al.
The Lancet. Neurology|August 16, 2025
Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trialThomas O Crawford, Laurent Servais, Eugenio Mercuri, et al.
The Lancet. Child & Adolescent Health|November 10, 2021
Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort studyClaudia Weiß, Andreas Ziegler, Lena-Luise Becker, et al.
Journal of Neuromuscular Diseases|December 4, 2022
Newbornscreening SMA - From Pilot Project to Nationwide Screening in GermanyWolfgang Müller-Felber, Astrid Blaschek, Oliver Schwartz, et al.
Journal of Neuromuscular Diseases|September 12, 2025
Phenotypic intrafamilial variability of 5q-associated spinal muscular atrophy: A systematic multicentre sibling studyBenedikt Becker, Isabell Cordts, Jutta Becker, et al.
Brain : a Journal of Neurology|July 20, 2022
Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophyAstrid Pechmann, Max Behrens, Katharina Dörnbrack, et al.
Journal of Neuromuscular Diseases|November 24, 2025
Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland-part I-systematic literature review and existing evidenceClaudia Weiß, Katharina Vill, Matthias Baumann, et al.
Pageof 6