Nadja Kaiser

Tuebingen, BW, Germany

Last Updated: 09/26/2026

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Found 11 publications

European expert recommendations for comprehensive pre-treatment, treatment-phase and post-treatment care of patients with metachromatic leukodystrophy treated with autologous haematopoietic stem and progenitor cell gene therapy.

European expert recommendations for comprehensive pre-treatment, treatment-phase and post-treatment care of patients with metachromatic leukodystrophy treated with autologous haematopoietic stem and progenitor cell gene therapy.

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

Published: March 25, 2026

Author Correction: Next-generation phenotyping integrated in a national framework for patients with ultrarare disorders improves genetic diagnostics and yields new molecular findings.

Author Correction: Next-generation phenotyping integrated in a national framework for patients with ultrarare disorders improves genetic diagnostics and yields new molecular findings.

Journal: Nature genetics

Published: June 24, 2025

Loss-of-function variants in the CAPN1 activator CD99L2 cause X-linked spastic ataxia.

Loss-of-function variants in the CAPN1 activator CD99L2 cause X-linked spastic ataxia.

Journal: Nature communications

Published: March 31, 2025

Clinical Effectiveness of Newborn Screening for Spinal Muscular Atrophy: A Nonrandomized Controlled Trial.

Clinical Effectiveness of Newborn Screening for Spinal Muscular Atrophy: A Nonrandomized Controlled Trial.

Journal: JAMA pediatrics

Published: April 08, 2024

Neurodevelopmental retardation and neurological symptoms in homozygous variegate porphyria: two new cases and a literature review.

Neurodevelopmental retardation and neurological symptoms in homozygous variegate porphyria: two new cases and a literature review.

Journal: Orphanet journal of rare diseases

Published: January 10, 2024

Next-generation phenotyping integrated in a national framework for patients with ultrarare disorders improves genetic diagnostics and yields new molecular findings.

Next-generation phenotyping integrated in a national framework for patients with ultrarare disorders improves genetic diagnostics and yields new molecular findings.

Journal: Nature genetics

Published: July 19, 2023

Hematopoietic Stem Cell Transplantation with Mesenchymal Stromal Cells in Children with Metachromatic Leukodystrophy.

Hematopoietic Stem Cell Transplantation with Mesenchymal Stromal Cells in Children with Metachromatic Leukodystrophy.

Journal: Stem cells and development

Published: March 24, 2022

Neurocognitive development in isolated Robin sequence treated with the Tuebingen palatal plate.

Neurocognitive development in isolated Robin sequence treated with the Tuebingen palatal plate.

Journal: Clinical oral investigations

Published: November 11, 2021

Early clinical course after hematopoietic stem cell transplantation in children with juvenile metachromatic leukodystrophy.

Early clinical course after hematopoietic stem cell transplantation in children with juvenile metachromatic leukodystrophy.

Journal: Molecular and cellular pediatrics

Published: March 04, 2020

Association of Age at Onset and First Symptoms With Disease Progression in Patients With Metachromatic Leukodystrophy.

Association of Age at Onset and First Symptoms With Disease Progression in Patients With Metachromatic Leukodystrophy.

Journal: Neurology

Published: February 05, 2020

Enlargement of peripheral nerves in Krabbe disease: The diagnostic value of nerve ultrasound.

Enlargement of peripheral nerves in Krabbe disease: The diagnostic value of nerve ultrasound.

Journal: Muscle & nerve

Published: August 20, 2019

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Last Updated: 09/26/2026