ArticleBone marrow transplantation2008
The controversial and changing role of haematopoietic cell transplantation for lysosomal storage disorders: an update.
Article in Bone marrow transplantation, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 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
20 citing papers in PubMed, 34 citations in OpenAlex.
- Fabry Disease: Molecular Basis, Pathophysiology, Diagnostics and Potential Therapeutic Directions.Biomolecules · 2021Review
- Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and Future.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2019Review
- Busulfan, Fludarabine, and Cyclophosphamide (BFC) conditioning allowed stable engraftment after haplo-identical allogeneic stem cell transplantation in children with adrenoleukodystrophy and mucopolysaccharidosis.Bone marrow transplantation · 2018Article
- New Approaches to Tay-Sachs Disease Therapy.Frontiers in physiology · 2018Review
- Induced pluripotent stem cell models of lysosomal storage disorders.Disease models & mechanisms · 2017Review
- Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?Journal of inherited metabolic disease · 2016Review
- Article
- Molecular Genetics and Metabolism Report Long-term follow-up of post hematopoietic stem cell transplantation for Hurler syndrome: clinical, biochemical, and pathological improvements.Molecular genetics and metabolism reports · 2015Article
- Therapies for the bone in mucopolysaccharidoses.Molecular genetics and metabolism · 2015Review
- Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome.Drug design, development and therapy · 2015Review
- Diagnostic and treatment strategies in mucopolysaccharidosis VI.The application of clinical genetics · 2015Review
- Gene therapy for the neurological manifestations in lysosomal storage disorders.Journal of lipid research · 2014Review
- Respiratory and sleep disorders in mucopolysaccharidosis.Journal of inherited metabolic disease · 2013Review
- Anaesthesia and airway management in mucopolysaccharidosis.Journal of inherited metabolic disease · 2013Review
- Recent gene therapy advancements for neurological diseases.Discovery medicine · 2013Review
- Course and management of allogeneic stem cell transplantation in patients with mitochondrial neurogastrointestinal encephalomyopathy.Journal of neurology · 2012Article
- Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI.Molecular genetics and metabolism · 2011Article
- Therapeutic liver repopulation for phenylketonuria.Journal of inherited metabolic disease · 2010Review
- Mucopolysaccharidosis VI.Orphanet journal of rare diseases · 2010Review
- Metachromatic leukodystrophy: an overview of current and prospective treatments.Bone marrow transplantation · 2008Review
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
Haematopoietic cell transplantation (HCT) can benefit some selected subsets of patients with lysosomal diseases. Results had been impressive in children with MPS I-H, but poor in other disorders. Careful, multidisciplinary decision-making regarding whether to recommend HCT and how to provide optimal pre- and post-HCT care has proven essential to increase the likelihood of a good outcome.
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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.