Klaus Goldhahn
Westend, BE, Germany
Last Updated: 09/26/2026
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Found 8 publications
Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland - part II - expert based recommendations for surveillance and management of side-effects.
Journal: Journal of neuromuscular diseases
Published: August 27, 2026
Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland-part I-systematic literature review and existing evidence.
Journal: Journal of neuromuscular diseases
Published: November 24, 2025
Risdiplam Add-On Therapy Following Onasemnogene Abeparvovec in Children With Spinal Muscular Atrophy and 2 SMN2 Copies: A Multi-Center Case Series.
Journal: Muscle & nerve
Published: July 23, 2025
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 study.
Journal: The Lancet regional health. Europe
Published: June 28, 2024
Use of Sodium Channel Blockers in the Thr226Met Pathologic Variant of SCN1A: A Case Report.
Journal: Neuropediatrics
Published: July 19, 2023
Closing the Gap - Detection of 5q-Spinal Muscular Atrophy by Short-Read Next-Generation Sequencing and Unexpected Results in a Diagnostic Patient Cohort.
Journal: Journal of neuromuscular diseases
Published: July 10, 2023
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 study.
Journal: The Lancet. Child & adolescent health
Published: July 19, 2021
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Last Updated: 09/26/2026