ArticleExperimental neurology2009
Promising CNS-directed enzyme replacement therapy for lysosomal storage diseases.
Article in Experimental neurology, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 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
13 citing papers in PubMed.
- An emerging phenotype of central nervous system involvement in Pompe disease: from bench to bedside and beyond.Annals of translational medicine · 2019Review
- Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease.Molecular therapy. Methods & clinical development · 2019Article
- Infantile Neuroaxonal Dystrophy: Diagnosis and Possible Treatments.Frontiers in genetics · 2018Article
- Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.Pediatric endocrinology reviews : PER · 2016Review
- Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis.Orphanet journal of rare diseases · 2016Review
- Pathogenesis and therapies for infantile neuronal ceroid lipofuscinosis (infantile CLN1 disease).Biochimica et biophysica acta · 2013Review
- Considerations for the treatment of infantile neuronal ceroid lipofuscinosis (infantile Batten disease).Journal of child neurology · 2013Article
- Biochemical evidence for superior correction of neuronal storage by chemically modified enzyme in murine mucopolysaccharidosis VII.Proceedings of the National Academy of Sciences of the United States of America · 2012Article
- Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy.Molecular genetics and metabolism · 2012Article
- Sialic acid deposition impairs the utility of AAV9, but not peptide-modified AAVs for brain gene therapy in a mouse model of lysosomal storage disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Large-volume intrathecal enzyme delivery increases survival of a mouse model of late infantile neuronal ceroid lipofuscinosis.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Article
- Lessons learnt from animal models: pathophysiology of neuropathic lysosomal storage disorders.Journal of inherited metabolic disease · 2010Article
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Abstract
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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.