rett-syndrome

Documents about rett-syndrome

• ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME#1007902ClinicalTrials.gov
• A Safety Study of NNZ-2566 in Pediatric Rett Syndrome#831478ClinicalTrials.gov
• A Safety Study of NNZ-2566 in Patients With Rett Syndrome#894547ClinicalTrials.gov
• Neurophysiologic Biomarkers in Rett Syndrome#870086ClinicalTrials.gov
• A Study to Evaluate Ketamine for the Treatment of Rett Syndrome#878775ClinicalTrials.gov
• Functional Abilities in Rett Syndrome#898197ClinicalTrials.gov
• Rett Syndrome Registry#894600ClinicalTrials.gov
• Analysis of the Glutathione Cycle in Children With Rett Syndrome#855757ClinicalTrials.gov
• GCB-002 in Treatment of Patients With Rett Syndrome#864582ClinicalTrials.gov
• A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome#195451ClinicalTrials.gov
• ANAVEX2-73 Study in Patients With Rett Syndrome#866909ClinicalTrials.gov
• Osteopathic Manipulative Treatment for Constipation in People With Rett Syndrome#967606ClinicalTrials.gov
• Natural History of Rett Syndrome & Related Disorders#992832ClinicalTrials.gov
• Biobanking of Rett Syndrome and Related Disorders#851205ClinicalTrials.gov
• Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)#913408ClinicalTrials.gov
• Leptin antagonism improves Rett syndrome phenotype in symptomatic Mecp2-deficient mice#231638HAL (France)
• Increased Osteoclast Activity Contributes to Bone Resorption and Osteopenia in a Rett Syndrome Mouse Model#239008HAL (France)
• Rare Diseases Clinical Research Network: Neurophysiological Correlates#838180ClinicalTrials.gov
• An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome#907693ClinicalTrials.gov
• Alteration of cholinergic pathway in the enteric nervous system of a mouse model of Rett syndrome#677064HAL (France)
• A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome#902384ClinicalTrials.gov
• Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome#993699ClinicalTrials.gov
• Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW#810062ClinicalTrials.gov
• Distribution of hand function by age in individuals with Rett syndrome#431471PubMed Central
• Impaired astrocyte-to-neuron cholesterol trafficking drives synaptic dysfunction in Rett syndrome#610848bioRxiv / medRxiv
• From Gene to Hope: Rett Syndrome and the Rise of Molecular Therapies#27541HAL (France)
• MeCP2 NID interaction with RNA: implications for Rett syndrome-relevant protein regulation.#145953NCBI PubMed Central
• Behavioral characteristics of individuals with Rett syndrome in Korea: A cross-sectional study.#250963NCBI PubMed Central
• Minimum data requirements and automated preprocessing for reliable EEG biomarkers in Rett syndrome.#327049NCBI PubMed Central
• Recent advances in epigenetic therapeutics for Rett syndrome: from mechanisms to clinical trials.#371526NCBI PubMed Central
• Mecp2 deficiency impairs microscale cortical network topology and dynamics in a Rett syndrome mouse model#300645Europe PMC
• Expanding the Phenotypic Spectrum of Raynaud–Claes Syndrome: A Rett-like Presentation with Two New Cases.#315751NCBI PubMed Central
• IGF1 peptide targets Rett Syndrome astrocytes to degrade IGF binding protein, rescue synaptogenesis and restore mitochondrial function#440222bioRxiv / medRxiv
• Characteristics of adult individuals with Rett syndrome treated or untreated with trofinetide in the United States.#446373NCBI PubMed Central
• Generation of four H1 hESC sublines carrying a hemizygous knock-out/mutant MECP2#955452Koc University Digital Collections
• Linguistic Validation of the Rett Syndrome Behavior Questionnaire Spanish Translation: a Two-Stage Caregiver Study Across Latin America#216678bioRxiv / medRxiv
• Single-nucleus profiling reveals a core disease signature and cell type-specific vulnerabilities in early Rett syndrome#273304Europe PMC
• Real-World Treatment Landscape of Individuals with Rett Syndrome Treated and Untreated with Trofinetide in the United States.#281047NCBI PubMed Central
• Translational reading frame predicts the pathogenicity of C-terminal frameshift deletions in MeCP2#669693Europe PMC
• Phage-assisted evolution of degron tags and botulinum neurotoxins for targeted protein degradation and delivery, alongside base editing for Rett syndrome#347573Harvard DASH
• Minimally Invasive Bipolar Technique for Scoliosis in Rett Syndrome—Results and Complications in a Series of 22 Cases#250369HAL (France)
• MeCP2-driven chromatin organization controls nuclear stiffness#479728TUdatalib System
• Obi Medical Robot: Evaluating Effectiveness Related to Usability#913146ClinicalTrials.gov
• MeCP2 heterochromatin organization is modulated by arginine methylation and serine phosphorylation#278225TUdatalib System
• Trofinetide Use and Treatment Patterns Among Children Aged 2–4 Years with Rett Syndrome in the United States: A Retrospective Specialty Pharmacy Linked Claims Database Analysis.#342636NCBI PubMed Central
• A Study of the Effectiveness and Safety of Risperidone Versus Placebo in the Treatment of Children With Autistic Disorder and Other Pervasive Developmental Disorders (PDD)#885803ClinicalTrials.gov
• Cyclin-dependent-like kinase 5 is required for pain signaling in human sensory neurons and mouse models#989950Spiral