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Batten disease

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Search Period All yearsLast 12 monthsLast 5 years Topic All topicsGeneralNutrition and dietAlternative treatment methods Type of research Any type of researchStrongest evidence only (29)Meta-analysis (6)Treatment guideline (3)Systematic review (10)Randomized trial (9)Phase 3 study (1)Clinical trial (58)Review article (128)Early study (phase 1 or 2) (21)Case report (105)Laboratory or animal research (71)Commentary or editorial (35) Studies currently recruiting participants only (24)

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Publications and studies (974)

• Patient advocate involvement in the design and conduct of breast cancer clinical trials requiring the collection of multiple biopsies. (2018/07/16) ♡
• LuTHy: a double-readout bioluminescence-based two-hybrid technology for quantitative mapping of protein-protein interactions in mammalian cells. (2018/07/11) ♡
• Corrected US opioid-involved drug poisoning deaths and mortality rates, 1999-2015. (2018/07/01) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Modulating membrane fluidity corrects Batten disease phenotypes in vitro and in vivo. (2018/07/01) ♡
• Approaches to parental demand for non-established medical treatment: reflections on the Charlie Gard case. (2018/07/01) ♡
• Global and Widespread Local White Matter Abnormalities in Juvenile Neuronal Ceroid Lipofuscinosis. (2018/07/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Progress in gene and cell therapies for the neuronal ceroid lipofuscinoses. (2018/07/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Clostridium difficile colitis complicating Kawasaki disease in children: Two case reports. (2018/06/13) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Novel compound heterozygous mutations causing Kufs disease type B. (2018/06/01) ♡
• The impact of a prolonged stay in the ICU on patients' fundamental care needs. (2018/06/01) ♡
• ISCEV extended protocol for the photopic On-Off ERG. (2018/06/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study of Intraventricular Cerliponase Alfa for CLN2 Disease. (2018/05/17) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Altered Cerebellar Short-Term Plasticity but No Change in Postsynaptic AMPA-Type Glutamate Receptors in a Mouse Model of Juvenile Batten Disease. (2018/05/17) ♡
• Loss of CLN7 results in depletion of soluble lysosomal proteins and impaired mTOR reactivation. (2018/05/15) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Juvenile-onset neuronal ceroid lipofuscinosis (CLN1) disease with a novel deletion and duplication in the PPT1 gene. (2018/05/15) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Prevention of Photoreceptor Cell Loss in a Cln6(nclf) Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar Cells. (2018/05/02) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Gene therapy: Gene therapy targets pathology in progranulin-deficient mice. (2018/05/01) ♡
• Characteristics of PPT1 and TPP1 enzymes in neuronal ceroid lipofuscinosis (NCL) 1 and 2 by dried blood spots (DBS) and leukocytes and their application to newborn screening. (2018/05/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Photosensitivity as an early marker of epileptic and developmental encephalopathies. (2018/05/01) ♡
• Neural stem cells for disease modeling and evaluation of therapeutics for infantile (CLN1/PPT1) and late infantile (CLN2/TPP1) neuronal ceroid lipofuscinoses. (2018/04/10) ♡
• Effect of treatment dose reductions in the setting of hand-foot syndrome on survival outcomes in patients with metastatic renal cell carcinoma treated with vascular endothelial growth factor receptor inhibitors. (2018/04/01) ♡
• Experimental infection of sheep, goats and cattle with a bluetongue virus serotype 4 field strain from Bulgaria, 2014. (2018/04/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Congenital Neuronal Ceroid Lipofuscinosis with a Novel CTSD Gene Mutation: A Rare Cause of Neonatal-Onset Neurodegenerative Disorder. (2018/04/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Videoconferencing for Management of Heart Failure: An Integrative Review. (2018/04/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Occipital epilepsy versus progressive myoclonic epilepsy in a patient with continuous occipital spikes and photosensitivity in electroencephalogram: A case report. (2018/04/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. The Retromer Complex and Sorting Nexins in Neurodegenerative Diseases. (2018/03/26) ♡
• Meta-analysisiAll studies on one question combined and calculated together. This is the strongest form of research that exists: a single loose study can be coincidence, dozens together much less so. The label says something about the design, not about the outcome — which can also be that something does NOT work. Timing of cognitive decline in CLN3 disease. (2018/03/01) ♡
• Evidence for Cholinergic Dysfunction in Autosomal Dominant Kufs Disease. (2018/03/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. [Autistic regression: clinical and aetiological aspects]. (2018/03/01) ♡
• Progranulin Gene Therapy Improves Lysosomal Dysfunction and Microglial Pathology Associated with Frontotemporal Dementia and Neuronal Ceroid Lipofuscinosis. (2018/02/28) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Altered Expression of Ganglioside Metabolizing Enzymes Results in GM3 Ganglioside Accumulation in Cerebellar Cells of a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis. (2018/02/22) ♡
• CRISPR/Cas9 in Leishmania mexicana: A case study of LmxBTN1. (2018/02/13) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Identification of two novel null variants in CLN8 by targeted next-generation sequencing: first report of a Chinese patient with neuronal ceroid lipofuscinosis due to CLN8 variants. (2018/02/08) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Inducible transgenic expression of tripeptidyl peptidase 1 in a mouse model of late-infantile neuronal ceroid lipofuscinosis. (2018/02/06) ♡
• Randomized researchiParticipants were divided into groups by lottery and compared with each other. This reduces the chance that a difference is due to something other than the treatment. A Community-Based, Bionic Leg Rehabilitation Program for Patients with Chronic Stroke: Clinical Trial Protocol. (2018/02/01) ♡
• GPR65 inhibits experimental autoimmune encephalomyelitis through CD4(+) T cell independent mechanisms that include effects on iNKT cells. (2018/02/01) ♡
• Metallothioneins are neuroprotective agents in lysosomal storage disorders. (2018/02/01) ♡
• CRISPR-Cas9-Mediated Correction of the 1.02 kb Common Deletion in CLN3 in Induced Pluripotent Stem Cells from Patients with Batten Disease. (2018/02/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Neuronal ceroid lipofuscinosis in Salukis is caused by a single base pair insertion in CLN8. (2018/02/01) ♡
• Cln5 is secreted and functions as a glycoside hydrolase in Dictyostelium. (2018/01/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. The strategic function of the P5-ATPase ATP13A2 in toxic waste disposal. (2018/01/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Neuronal ceroid lipofuscinosis with severe biventricular impairment: a rare genetic disorder with associated myopathy? (2018/01/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Gene Therapy Approaches to Treat the Neurodegeneration and Visual Failure in Neuronal Ceroid Lipofuscinoses. (2018/01/01) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Further Characterization of the Predominant Inner Retinal Degeneration of Aging Cln3 (Δex7/8) Knock-In Mice. (2018/01/01) ♡
• Progranulin-mediated deficiency of cathepsin D results in FTD and NCL-like phenotypes in neurons derived from FTD patients. (2017/12/15) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Dysregulation of autophagy as a common mechanism in lysosomal storage diseases. (2017/12/12) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Canine neuronal ceroid lipofuscinoses: Promising models for preclinical testing of therapeutic interventions. (2017/12/01) ♡
• Breastfeeding Duration and Adolescent Educational Outcomes: Longitudinal Evidence From India. (2017/12/01) ♡
• Lack of specificity of antibodies raised against CLN3, the lysosomal/endosomal transmembrane protein mutated in juvenile Batten disease. (2017/11/23) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Selective depletion of microglial progranulin in mice is not sufficient to cause neuronal ceroid lipofuscinosis or neuroinflammation. (2017/11/17) ♡

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