Research map: Haematopoetic stem and progenitor cell gene therapy for metachromatic leukodystrophy: Mission possible!

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Papers in this map

  1. Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy · Christine Duncan · 2024 · 142 citations · Cited by this paper
  2. Seizures in children and adolescents with high-grade glioma: a retrospective, monocentric analysis · 2025 · Related
  3. Metachromatic Leukodystrophy · Marije A B C Asbreuk · 2025 · 12 citations · Cited by this paper
  4. Diagnostic value of genetic testing, with focus on CACNA1A, in children with episodic neurologic disorders: a single-centre retrospective study · 2025 · Related
  5. Treatment of leukodystrophies: Advances and challenges · Nicole I. Wolf · 2025 · 10 citations · Cited by this paper
  6. Artificial intelligence in pediatric stroke: An ally in screening? · 2026 · Related
  7. Key lessons from the first international treatment eligibility committee: the case of metachromatic leukodystrophy · Daphne H. Schoenmakers · 2025 · 7 citations · Cited by this paper
  8. Complex febrile seizures: urgent need to reconsider existing guidelines – how soon is now? · 2026 · Related
  9. Gene therapy in neuronopathic lysosomal storage disorders · Aimée Donald · 2025 · 3 citations · Cited by this paper
  10. An objective and simple aEEG parameter for predicting neurological outcomes in extremely preterm infants · 2026 · Related
  11. Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations · Lucas Tricoli · 2025 · 1 citation · Cited by this paper
  12. Genotypic and phenotypic spectrum of congenital myasthenic syndrome: Insights from Southeastern Türkiye · 2026 · Related
  13. 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 · Lucia Laugwitz · 2026 · 1 citation · Cited by this paper
  14. Incidence and timing of diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2): A nationwide study using the French hospital discharge database · 2025 · Related
  15. In search of “what really matters”: Insights from a web-based survey on Patient-Centered Outcomes in GLUT1DS · 2026 · Related
  16. Clinical spectrum and genetic landscape of duchenne muscular dystrophy in Azerbaijan · 2026 · Related

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