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Rare and hereditary disorders
Batten disease
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Publications and studies (974)
- 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. Progressive Myoclonic Epilepsies - A Pragmatic Review. (2026/03/01) ♡
- A Flupirtine Benzyl Carbamate Improves Neurocognitive Deficits and Molecular Pathology in the Cln6(nclf) Mouse. (2026/02/28) ♡
- 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. A roadmap for a patient-centred approach to Pompe disease management. (2026/02/14) ♡
- IFN-γ-induced trained immunity enhances killing of priority pathogens in healthy and genetically vulnerable individuals. (2026/02/10) ♡
- Neurodevelopmental and Psychiatric Disorders and the Use of Psychotropic Medications in a National Sample of Individuals With Juvenile Neuronal Ceroid Lipofuscinosis. (2026/02/05) ♡
- Bi-allelic TPP1 variants in neuronal ceroid lipofuscinosis 2: clinical findings from an Iranian cohort of 20 patients, founder effect, and in silico analyses. (2026/02/02) ♡
- Dysfunction of GABAergic interneurons underlies altered neural network oscillations associated with epileptiform activity in PPT1-deficient mice. (2026/02/02) ♡
- Age at onset and gene variants predict lifespan and disease duration in childhood neuronal ceroid lipofuscinoses. (2026/02/01) ♡
- Natural history and variants in neuronal ceroid lipofuscinoses: Uncoupling genotype and phenotype. (2026/02/01) ♡
- Lysosomal TRPML1 activation modulates synaptic transmission and intrinsic neuronal excitability. (2026/02/01) ♡
- Progressive rod bipolar cell loss, ubiquitin accumulation, and subretinal drusenoid deposits in Cln3Δex7/8 mice: New insights into juvenile neuronal ceroid lipofuscinosis pathogenesis. (2026/02/01) ♡
- An exercise intervention in children and young adults with McArdle disease: feasibility, acceptability, and clinical outcomes. (2026/01/31) ♡
- High-resolution promoter interaction analysis implicates genes involved in the activation of Type 3 Innate Lymphoid Cells in autoimmune disease risk. (2026/01/10) ♡
- Comparison of whole genome sequencing approaches for Capripox viruses. (2026/01/08) ♡
- 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. A timeline of symptom onset and disease progression in CLN3 disease. (2026/01/07) ♡
- Delayed Aggressive Local Recurrence of Chondroblastic Osteosarcoma 3 Years After an Excellent Pathological Response: A Case Report. (2026/01/07) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Unraveling Neuronal Ceroid Lipofuscinosis: Insights From Two Pediatric Cases in Peripheral India. (2026/01/01) ♡
- Application of green nanoparticles in dental materials: A scoping review. (2026/01/01) ♡
- 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. CLN2 Disease: Current Understandings, Challenges, and Future Directions. (2026/01/01) ♡
- Determining Lysosomal Enzyme Activity Using Fluorogenic Probes. (2026/01/01) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Bioenergetic Profiling Applied to a Zebrafish Model of Neuronal Ceroid Lipofuscinosis, a Lysosomal Storage Disorder. (2026/01/01) ♡
- 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. First-in-human high dose AAV9 intrathecal gene therapy for paediatric CLN7 disease: a phase 1, open-label, single ascending dose, non-randomised clinical trial. (2026/01/01) ♡
- Incidence and timing of diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2): A nationwide study using the French hospital discharge database. (2026/01/01) ♡
- Sunken eyes as a peculiar finding in neuronal ceroid lipofuscinoses. (2026/01/01) ♡
- 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. Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol) (2026-07-31) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Caregiving Networks Across Disease Context and the Life Course (2026-07-30) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Cerliponase Alfa Observational Study in the US (2026-06-30) ♡
- 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 CLN3 Batten Disease (2026-06-10) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical Controls (2026-06-05) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Inherited Retinal Degenerative Disease Registry (2026-05-19) ♡
- 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 Trial for CLN6 Batten Disease (2026-05-14) ♡
- Phase 3 studyiResearch in a large group of patients, the final step before a treatment can be approved. What comes out here carries significant weight. Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3 (2026-05-08) ♡
- 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. Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2 (2026-04-30) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients (2026-04-08) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Italian NCL Registry: a Registry for NCL as an Integration Tool for Future Therapeutic Strategies (2026-03-27) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Investigations of Juvenile Neuronal Ceroid Lipofuscinosis (2026-02-24) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Longitudinal Study of Neurodegenerative Disorders (2026-02-09) ♡
- First Reported Case of CLN5 Disease in Japan: Identification of a Novel Homozygous Pathogenic Variant Through Whole Genome Sequencing. (2025/12/30) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Limited therapeutic efficacy of N-acetyl-L-leucine in a mouse model of CLN1 disease. (2025/12/29) ♡
- 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. Safety and efficacy of combining midostaurin and gemtuzumab ozogamicin with induction chemotherapy in FLT3-mutated AML. (2025/12/23) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Enteric nervous system degeneration in human and murine CLN3 disease, is ameliorated by gene therapy in mice. (2025/12/22) ♡
- Protocol for screening of small molecules in a CLN3 disease patient-specific iPSC-derived neuronal progenitor cell model. (2025/12/19) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Gastrointestinal symptoms in neuronal ceroid lipofuscinoses (NCLs): an observational study on prevalence, timing of progression, and impact on quality of life. (2025/12/11) ♡
- Prodromal pathogenesis of CLN7 Batten Disease revealed by multimodal biomarkers in macaques. (2025/12/05) ♡
- Loss of the lysosomal protein CLN3 triggers c-Abl-dependent YAP1 pro-apoptotic signaling. (2025/12/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Persistent lymphopenia in a Japanese boy with neuronal ceroid lipofuscinosis type 3. (2025/12/01) ♡
- Prime editing-installed suppressor tRNAs for disease-agnostic genome editing. (2025/12/01) ♡
- Modification and Validation of a Reference Real-Time RT-PCR Method for the Detection of a New African Horse Sickness Virus Variant. (2025/11/25) ♡
- Longitudinal Exploration of Auditory Sensory-Perceptual Processing in CLN3 Disease (Juvenile Neuronal Ceroid Lipofuscinosis (Batten disease)): A High-Density Auditory Evoked Potential (AEP) Study. (2025/11/20) ♡
- The missing piece: Solving the 50-year puzzle of BMP synthesis in neurodegeneration. (2025/11/13) ♡
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