ReviewMolecular diagnosis & therapy2021
Elivaldogene Autotemcel: First Approval.
Review in Molecular diagnosis & therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
25 citing papers in PubMed, 40 citations in OpenAlex.
- Attitudes Toward Sex-Specific Versus Universal Newborn Screening for X-Linked Adrenoleukodystrophy in Hong Kong.International journal of neonatal screening · 2026Article
- Neurodegenerative Diseases in Children: A Comprehensive Review.International journal of molecular sciences · 2026Review
- Pompe Disease: Pathogenesis, Molecular Mechanisms, Neurological Aspects, Diagnostics and Modern Therapeutic Approaches.International journal of molecular sciences · 2026Review
- From lipid function to dysfunction: Very long-chain fatty acids as emerging regulators of neuroinflammatory pathways.iScience · 2026Review
- Progress in peptide and protein therapeutics: Challenges and strategies.Acta pharmaceutica Sinica. B · 2025Review
- Mosaic X-linked adrenoleukodystrophy in males identified by newborn screening and next-generation sequencing.NPJ genomic medicine · 2025Article
- Rewriting the script: gene therapy and genome editing for von Willebrand Disease.Frontiers in genome editing · 2025Review
- Skeleton keys and Trojan horses: a review of therapeutic delivery to the brain.Frontiers in cell and developmental biology · 2025Review
- Inherited metabolic epilepsies-established diseases, new approaches.Epilepsia open · 2024Review
- Advances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders.Molecular therapy. Methods & clinical development · 2024Review
- From gene to therapy: a review of deciphering the role of ABCD1 in combating X-Linked adrenoleukodystrophy.Lipids in health and disease · 2024Review
- Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Tumor Microenvironment-Responsive Nanocapsule Delivery CRISPR/Cas9 to Reprogram the Immunosuppressive Microenvironment in Hepatoma Carcinoma.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2024Article
- Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease.Nature medicine · 2023Article
- Is it enough just to demonstrate that the advanced therapy medicinal products do work or we would prefer to keep walking on the Moon?Regenerative therapy · 2023Article
- Clinical and biochemical footprints of inherited metabolic diseases. XV. Epilepsies.Molecular genetics and metabolism · 2023Review
- Gene and Cellular Therapies for Leukodystrophies.Pharmaceutics · 2023Review
- Slow and steady wins the race: Optimizing lentiviral vector production for efficient clinical-scale manufacturing.Molecular therapy. Methods & clinical development · 2023Article
- Innate Immune Response to Viral Vectors in Gene Therapy.Viruses · 2023Review
- Advancing cell therapy for neurodegenerative diseases.Cell stem cell · 2023Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Elivaldogene autotemcel (SKYSONA™, eli-cel; Lenti-D™ gene therapy) is a gene therapy that has been developed by bluebird bio for the treatment of cerebral adrenoleukodystrophy (CALD), a rare, X-linked genetic disease that mainly affects the nervous system and adrenal glands. In July 2021, elivaldogene autotemcel received its first approval (in the EU) for the treatment of for the treatment of early CALD in patients < 18 years of age, with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling haematopoietic stem cell (HSC) donor is not available. This article summarizes the milestones in the development of elivaldogene autotemcel leading to this first approval.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.