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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)

• Benchmarking Nanopore Sequencing for CLN2 (TPP1) Mutation Detection: Integrating Rapid Genomics and Orthogonal Validation for Precision Diagnostics. (2025/05/23) ♡
• 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. Neurodegenerative Disease and Association Football (NDAF): Systematic Review and Meta-Analysis. (2025/05/21) ♡
• Evidence of the impact of CLN2 and CLN3 Batten disease on families in the United Kingdom. (2025/05/12) ♡
• Niemann Pick C1 mistargeting disrupts lysosomal cholesterol homeostasis contributing to neurodegeneration in a Batten disease model. (2025/05/09) ♡
• 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 type 11 in early childhood. (2025/05/02) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Expanded Phenotype of the Cln6(nclf) Mouse Model. (2025/04/30) ♡
• Novel surgical approach for intraventricular cerliponase alfa enzyme replacement therapy via central venous access device (CVAD) port in neuronal ceroid lipofuscinosis type 2 (CLN2) disease. (2025/04/29) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Intravitreal Enzyme Replacement Therapy Slows Retinopathy in Late Infantile Ceroid Lipofuscinosis Type 2. (2025/04/01) ♡
• Magnetic Resonance Imaging as a Readout of CLN5 Gene Therapy Efficacy in Sheep. (2025/04/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. A Rare Case Report of Neurodegenerative Disease With Oro-Dental Trauma. (2025/03/30) ♡
• Syndromic forms of inherited retinal dystrophies: a comprehensive molecular diagnosis of consanguineous Pakistani families using capture panel sequencing. (2025/03/26) ♡
• 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. Enzyme Replacement Therapy in CLN2-Associated Retinopathy. (2025/03/01) ♡
• The Wechsler Intelligence Scale for Children, fourth and fifth editions perform comparably in children with Batten Disease. (2025/02/27) ♡
• TRAM-LAG1-CLN8 family proteins are acyltransferases regulating phospholipid composition. (2025/02/21) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Purifying and profiling lysosomes to expand understanding of lysosomal dysfunction-associated diseases. (2025/02/17) ♡
• Clinical disease in British sheep infected with an emerging strain of bluetongue virus serotype 3. (2025/02/15) ♡
• Systematic reviewiAll research on one question is searched according to fixed rules and compared side by side, so that no studies with poor outcomes are missed. The use of nanocarriers in treating Batten disease: A systematic review. (2025/02/10) ♡
• Activation of D2-like dopamine receptors improves the neuronal network and cognitive function of PPT1KI mice. (2025/02/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. CLN6-related continuum phenotype caused by aberrant splicing. (2025/02/01) ♡
• 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. Neuronal Ceroid Lipofuscinosis-Concepts, Classification, and Avenues for Therapy. (2025/02/01) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. The Batten disease gene Cln3 is required for the activation of intestinal stem cell during regeneration via JAK/STAT signaling in Drosophila. (2025/01/23) ♡
• 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 Role of Visual Electrophysiology in Systemic Hereditary Syndromes. (2025/01/23) ♡
• Genetic spectrum of neuronal ceroid lipofuscinosis & its genotype-phenotype correlation -A single centre experience of 56 cases. (2025/01/15) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Neuronal ceroid lipofuscinosis 11 (CLN11) presenting with early-onset cone-rod dystrophy and learning difficulties. (2025/01/15) ♡
• Gene therapy ameliorates bowel dysmotility and enteric neuron degeneration and extends survival in lysosomal storage disorder mouse models. (2025/01/15) ♡
• Two-year follow-up of gait and postural control following initiation of recombinant human tripeptidyl intracerebroventricular enzyme replacement therapy in two atypical CLN2 patients. (2025/01/07) ♡
• Open-label evaluation of oral trehalose in patients with neuronal ceroid lipofuscinoses. (2025/01/07) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Trehalose Ameliorates Zebrafish Emotional and Social Deficits Caused by CLN8 Dysfunction. (2025/01/05) ♡
• 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. Adeno-associated virus vectors and neurotoxicity-lessons from preclinical and human studies. (2025/01/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Further description of the phenotypic spectrum of neuronal ceroid lipofuscinosis type 11. (2025/01/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Drug-refractory epilepsy due to a novel CLN5 mutation: A report of three patients from an Indian family. (2025/01/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Clinical, Pathological, and Molecular Findings in a Mexican Patient With Neuronal Ceroid Lipofuscinosis Type 2: Support for Pathogenicity of the c.1226 G>T Variant and for Presence of Cherry-Red Spot in This Disease. (2025/01/01) ♡
• Speech, Language and Non-verbal Communication in CLN2 and CLN3 Batten Disease. (2025/01/01) ♡
• 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. Milasen: The Emerging Era of Patient-Customized N-of-1 Antisense Oligonucleotides as Therapeutic Agents for Genetic Diseases. (2025/01/01) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. Study for the Treatment for CLN7 Disease (2025-12-31) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease (2025-12-18) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease (2025-10-24) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease (2025-10-24) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer (2025-10-02) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Clinical and Neuropsychological Investigations in Batten Disease (2025-09-12) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease (2025-09-09) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells (2025-09-08) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Baby Detect : Genomic Newborn Screening (2025-08-12) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Natural History Study of Batten Disease (2025-07-30) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Disease (2025-07-30) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (2025-05-29) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. NYSCF Scientific Discovery Biobank (2025-03-03) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease (2025-02-17) ♡
• Tagless LysoIP for immunoaffinity enrichment of native lysosomes from clinical samples. (2024/12/26) ♡
• Neuronal ceroid lipofuscinoses type 7 (CLN7): a case series reporting cross sectional and retrospective clinical data to evaluate validity of standardized tools to assess disease progression, quality of life, and adaptive skills. (2024/12/19) ♡

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