genetic-diseases

Documents about genetic-diseases

• Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg#913184ClinicalTrials.gov
• A Trial of Metformin in Individuals With Fragile X Syndrome#290960ClinicalTrials.gov
• The disease burden and health-related quality of life in Chinese children with genetic cholestatic liver diseases: a cross-sectional study of progressive familial intrahepatic cholestasis and Alagille syndrome.#295477NCBI PubMed Central
• A Trial of Metformin in Individuals With Fragile X Syndrome (Met)#633857ClinicalTrials.gov
• Hormonal and Environmental Risk Factors for Developing Systemic Lupus Erythematosus: The Carolina Lupus (CLU) Study#853317ClinicalTrials.gov
• Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy#868227ClinicalTrials.gov
• Warfarin Anticoagulation in Patients in Sub-Saharan Africa#872671ClinicalTrials.gov
• Coping Strategies, Loneliness, and Resilience in Patients With Dermatosis#888807ClinicalTrials.gov
• Genetic predisposition and biomarkers in polycystic ovary syndrome among Filipino women: A systematic review#210442Animo Repository
• Stage-specific associations of mineralization markers with CKM syndrome: Nationwide survey and genetic evidence for Alkaline phosphatase’s unique clinical role#297783PLOS
• Oxandrolone Compared With a Placebo on Growth Rate in Girls With Growth Hormone-Treated Turner's Syndrome#848863ClinicalTrials.gov
• Phase III Randomized Study of Poloxamer 188 for Vaso-Occlusive Crisis of Sickle Cell Disease#870000ClinicalTrials.gov
• SMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)#846743ClinicalTrials.gov
• Molecular characterization of Theileria species and Theileria parva genetic diversity associated with bovine theileriosis outbreaks in Zimbabwe#179458HAL (France)
• Clinical Utility Of Genetic Screening For HLA-B*1301, On Susceptibility To Dapsone Hypersensitivity Syndrome#865735ClinicalTrials.gov
• Sonrotoclax and BCMA Bispecific Antibody in Newly Diagnosed Systemic AL Amyloidosis Based on t(11;14) Genetic Stratification#982262ClinicalTrials.gov
• Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa#841708ClinicalTrials.gov
• Study of the Pathophysiology of RNU4ATAC and RTTN Associated Syndromes#911109ClinicalTrials.gov
• Identification and molecular characterization of Mycobacterium bovis DNA in GeneXpert® MTB/RIF ultra-positive, culture-negative sputum from a rural community in South Africa#225370SUNScholar
• Tailoring Genetic Approaches for Easier Detection of Anti-3.7 Alpha-Globin Gene Triplication in the Iranian Population#624485Figshare
• Fluorodeoxyglucose-Positron Emission Tomography (FDG-PET) to Evaluate Autoimmune Lymphoproliferative Syndrome (ALPS) and ALPS-associated Lymphoma#832097ClinicalTrials.gov
• Genetic analysis of non‐syndromic craniosynostosis#970656eScholarship
• Phase II Study of the Multichannel Auditory Brain Stem Implant for Deafness Following Surgery for Neurofibromatosis 2#1008943ClinicalTrials.gov
• Detection and Characterization of Infections and Infection Susceptibility#863298ClinicalTrials.gov
• Identification of Genetic Causes of Calcific Aortic Valve Disease#894085ClinicalTrials.gov
• Integrating structure to protein-protein interaction networks that drive metastasis to brain and lung in breast cancer#954521Koc University Digital Collections
• Assessing the genetic architecture of epithelial ovarian cancer histological subtypes#612070eScholarship
• Cross-family and phage-specific gene requirements for Klebsiella infection revealed by scalable RB-TnSeq genetic screens.#624393eScholarship
• Use Massive Parallel Sequencing and Exome Capture Technology to Sequence the Exome of Fanconi Anemia Children and Their Patents#918332ClinicalTrials.gov
• Genetic Testing of CYP2C19 in Prognostic Evaluation of Long-Term Major Adverse Cardiac and Vascular Events#868583ClinicalTrials.gov
• Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients#966529ClinicalTrials.gov
• Social and Genetic Networks of HIV-1 Transmission in New York City#491877eScholarship
• Evaluation of Patients With Immune Function Abnormalities#881950ClinicalTrials.gov
• Turkish Society of Cardiology consensus report on recommendations for athletes with high-risk genetic cardiovascular diseases or implanted cardiac devices#955472Koc University Digital Collections
• Raw Data Supporting: Area-Wide Suppression of the Mediterranean Fruit Fly (Ceratitis capitata) Using a Male-Only Sterile Insect Technique Program in Citrus Orchards of Western Türkiye#361730Figshare
• Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders#893540ClinicalTrials.gov