Andrea Klein

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

Cerebellar Atrophy in Pediatric-Onset Opsoclonus-Myoclonus-Ataxia Syndrome: A Large International Cohort Study.

Journal: Neurology(R) neuroimmunology & neuroinflammation

Published: July 16, 2026

Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial.

Journal: The Lancet. Child & adolescent health

Published: January 28, 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

NEP 50th Annual Meeting of the Society for Neuropediatrics 2025.

Journal: Neuropediatrics

Published: September 26, 2025

283rd ENMC international workshop: Establishing expert care recommendations for LAMA2-RD: A prototype for the development of congenital muscular dystrophy subtype-specific care guidelines. Hoofddorp, The Netherlands, January 17th-19th 2025.

Journal: Neuromuscular disorders : NMD

Published: September 12, 2025

A20 (TNFAIP3) Distinguishes Attack From Remission in Pediatric Patients With Monophasic MOGAD.

Journal: Neurology(R) neuroimmunology & neuroinflammation

Published: August 18, 2025

Intracranial Rescue Stenting in Pediatric Focal Cerebral Arteriopathy.

Journal: Clinical neuroradiology

Published: May 02, 2025

Gene therapy approval for Duchenne muscular dystrophy: a European perspective.

Journal: Lancet (London, England)

Published: April 03, 2025

Real-world data on the effect of long-term treatment with nusinersen over > 4 years in a cohort of Swiss patients with spinal muscular atrophy.

Journal: Clinical neurology and neurosurgery

Published: February 18, 2025

Impact of lesion metrics and neurological functions on long-term cognitive outcome in childhood stroke.

Journal: European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society

Published: December 17, 2024

Education and participation in children and adolescents with Duchenne muscular dystrophy in Switzerland.

Journal: European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society

Published: October 15, 2024

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.

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.

Cerebellar Atrophy in Pediatric-Onset Opsoclonus-Myoclonus-Ataxia Syndrome: A Large International Cohort Study.

Cerebellar Atrophy in Pediatric-Onset Opsoclonus-Myoclonus-Ataxia Syndrome: A Large International Cohort Study.

Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial.

Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial.

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.

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.

NEP 50th Annual Meeting of the Society for Neuropediatrics 2025.

NEP 50th Annual Meeting of the Society for Neuropediatrics 2025.

283rd ENMC international workshop: Establishing expert care recommendations for LAMA2-RD: A prototype for the development of congenital muscular dystrophy subtype-specific care guidelines. Hoofddorp, The Netherlands, January 17th-19th 2025.

283rd ENMC international workshop: Establishing expert care recommendations for LAMA2-RD: A prototype for the development of congenital muscular dystrophy subtype-specific care guidelines. Hoofddorp, The Netherlands, January 17th-19th 2025.

A20 (TNFAIP3) Distinguishes Attack From Remission in Pediatric Patients With Monophasic MOGAD.

A20 (TNFAIP3) Distinguishes Attack From Remission in Pediatric Patients With Monophasic MOGAD.

Intracranial Rescue Stenting in Pediatric Focal Cerebral Arteriopathy.

Intracranial Rescue Stenting in Pediatric Focal Cerebral Arteriopathy.

Gene therapy approval for Duchenne muscular dystrophy: a European perspective.

Gene therapy approval for Duchenne muscular dystrophy: a European perspective.

Real-world data on the effect of long-term treatment with nusinersen over > 4 years in a cohort of Swiss patients with spinal muscular atrophy.

Real-world data on the effect of long-term treatment with nusinersen over > 4 years in a cohort of Swiss patients with spinal muscular atrophy.

Impact of lesion metrics and neurological functions on long-term cognitive outcome in childhood stroke.

Impact of lesion metrics and neurological functions on long-term cognitive outcome in childhood stroke.

Education and participation in children and adolescents with Duchenne muscular dystrophy in Switzerland.

Education and participation in children and adolescents with Duchenne muscular dystrophy in Switzerland.

Real-world data on the effect of long-term treatment with nusinersen over > 4 years in a cohort of Swiss patients with spinal muscular atrophy.

Real-world data on the effect of long-term treatment with nusinersen over > 4 years in a cohort of Swiss patients with spinal muscular atrophy.