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About 87,528 results

Immunoglobulin A nephropathy in pediatrics: an up-to-date.
https://doi.org/10.1111/nep.13987
Nephrology (Carlton, Vic.); Paranhos RM, de Souza Figueiredo GA et. al.

Oct 23rd, 2021 - Immunoglobulin A nephropathy is the main cause of glomerulonephritis globally and an important etiology of end-stage renal disease in children. It has been considered an autoimmune disease that can lead to the production of autoantibodies against abnormal IgA1 and formation of immune complexes. These autoantibodies and immune complexes deposit in the glomeruli, resulting in renal injury. At the...

A modern approach to dyslipidemia.
https://doi.org/10.1210/endrev/bnab037
Endocrine Reviews; Berberich AJ, Hegele RA

Oct 23rd, 2021 - Lipid disorders involving derangements in serum cholesterol, triglycerides or both are commonly encountered in clinical practice and often have implications for cardiovascular risk and overall health. Recent advances in knowledge, recommendations and treatment options have necessitated an updated approach to these disorders. Older classification schemes have outlived their usefulness yielding t...

Injectable Hypomethylating Agents for Management of Myelodysplastic Syndromes: Patients...
https://doi.org/10.1016/j.clml.2021.09.009
Clinical Lymphoma, Myeloma & Leukemia; Zeidan AM, Jayade S et. al.

Oct 23rd, 2021 - Until recently, patients with MDSs could receive HMAs via intravenous (IV) or subcutaneous (SC) administration. An oral HMA was recently approved as an alternative to IV/SC administration. This study assessed the impact of IV/SC HMA on MDS patients, and their experience of, challenges with, and views about oral MDS treatment. We conducted an online cross-sectional survey among adult MDS patient...

The Expanding Role of Pyridine and Dihydropyridine Scaffolds in Drug Design.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8520849
Drug Design, Development and Therapy; Ling Y, Hao ZY et. al.

Oct 23rd, 2021 - Pyridine-based ring systems are one of the most extensively used heterocycles in the field of drug design, primarily due to their profound effect on pharmacological activity, which has led to the discovery of numerous broad-spectrum therapeutic agents. In the US FDA database, there are 95 approved pharmaceuticals that stem from pyridine or dihydropyridine, including isoniazid and ethionamide (t...

Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
https://clinicaltrials.gov/ct2/show/NCT01306019

Oct 22nd, 2021 - This is a Phase I/II non-randomized clinical trial of ex vivo hematopoietic stem cell (HSC) gene transfer treatment for X-linked severe combined immunodeficiency (XSCID, also known as SCID-X1) using a self-inactivating lentiviral vector incorporating additional features to improve safety and performance. The study will treat 13 patients with XSCID who are between 2 and 40 years of age and who h...

Phase 1a/1b Study of TPST-1495 as a Single Agent and in Combination With Pembrolizumab in Subjects With Solid Tumors
https://clinicaltrials.gov/ct2/show/NCT04344795

Oct 22nd, 2021 - This is a first-in-human Phase 1a/1b, multicenter, open-label, dose-escalation, dose and schedule optimization, and expansion study of TPST-1495 as a single agent and in combination with pembrolizumab to determine its MTD, safety, tolerability, pharmacokinetics (PD), pharmacodynamics (PK) and preliminary anti-tumor activity in subjects with advanced solid tumors. Subjects with all histologic ty...

Efficacy and Safety of Mydriatic Microdrops for Retinopathy Of Prematurity Screening
https://clinicaltrials.gov/ct2/show/NCT05043077

Oct 22nd, 2021 - A non-inferiority, crossover, randomized controlled trial will be conducted for this purpose. Participants will be randomly assigned to receive either a) microdrops on their first and standard drops on their second screening examination a week later (M/S group), or b) standard drops first and microdrops a week later (S/M group). Microdrops (6.5 μL) will be instilled using a calibrated micropipe...

Second-line Treatment of Primary Autoimmune Hemolytic Anemia
https://clinicaltrials.gov/ct2/show/NCT05057468

Oct 22nd, 2021 - Auto-immune hemolytic anemia (AIHA) is a heterogeneous syndrome in adults. This disease is associated with significant morbidity-mortality. First-line treatment with prolonged corticosteroid is well identified but there is not enough data about cyclosporine treatment in case of resistance or dependence on steroids. the cyclosporine showed efficacy in many immune cytopenic diseases in the light ...

T Cell Receptor Gene Therapy Targeting KK-LC-1 for Gastric, Breast, Cervical, Lung and Other KK-LC-1 Positive Epithelial Cancers
https://clinicaltrials.gov/ct2/show/NCT05035407

Oct 22nd, 2021 - Background: KK-LC-1 is a cancer germline (CG) antigen with expression restricted to germ cells (which lack MHC class I expression) in adults and epithelial cancers including lung, breast and gastric. This limited expression pattern makes it an ideal target for T Cell Receptor (TCR) gene therapy. TCR T cell therapy targeting CG antigens has been shown to induce objective responses without autoim...

Gabapentin for Restoring GABA/Glutamate Homeostasis in Co-occurring Bipolar and Cannabis Use Disorders
https://clinicaltrials.gov/ct2/show/NCT05064319

Oct 22nd, 2021 - This research study evaluates the effects of an FDA-approved medication Gabapentin in individuals with Bipolar Disorder who smoke marijuana. Participants in the study will will be assigned to take either Gabapentin or a matched placebo. Study medication will be taken for 17 days. There will be 5 study visits, with 2 MRI brain imaging scans completed. Questionnaires and clinical interview measur...

A Study to Investigate the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder
https://clinicaltrials.gov/ct2/show/NCT05064878

Oct 22nd, 2021 - This is a 2-part multicenter trial. Part 1 is a 20-week randomized, double-blind, placebo-controlled, fixed-dose, parallel-group study to examine the efficacy and safety of ZX008 as an adjunctive therapy (to existing concomitant treatment with antiepileptic treatments [AETs]) in children and adults with a CDD diagnosis and uncontrolled seizures. Part 1 of the study is 20 weeks in duration and w...

Maintenance Ipilimumab + Nivolumab Post Induction Chemotherapy + SBRT for First Line Treatment Stage IV Pancreatic Cancer
https://clinicaltrials.gov/ct2/show/NCT05088889

Oct 22nd, 2021 - This is a single arm, single institution study, which aims to evaluate the efficacy and safety of combination therapy with radiosurgery nivolumab and ipilimumab as a maintenance regimen following first line induction chemotherapy in patients with metastatic pancreatic cancer. Chemotherapy will be administrated for a minimum of 4 cycles. Patients who have not progressed on chemotherapy will rece...

A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome
https://clinicaltrials.gov/ct2/show/NCT05027945

Oct 22nd, 2021 - Background: In 2019, investigators at the National Institutes of Health defined a new disease syndrome named VEXAS: Vacuoles in bone marrow cells, E1 enzyme mutations, X-linked, Autoinflammatory, Somatic syndrome. This syndrome is characterized by inflammatory and hematologic features and is frequently accompanied by marrow dysplasia, progressive bone marrow failure, and in some cases, the deve...

Utility of 18FDOPA PET/MRI for Focal Hyperinsulinism
https://clinicaltrials.gov/ct2/show/NCT05088798

Oct 22nd, 2021 - Congenital hyperinsulinism (CHI) is a clinically and genetically heterogeneous disorder that is the most common cause of permanent hypoglycemia in infants and children. More than 50% of the medically-unresponsive patients have focal disease. Which is characterized by a distinct region of Beta-cell hyperplasia in the pancreas, due to a somatic loss of a gene that regulates cell proliferation. Re...

Growth Hormone Pretreatment in Poseidon Type IV Undergoing ICSI Using Minimal Induction Protocol: A Randomized Controlled Trial
https://clinicaltrials.gov/ct2/show/NCT05089344

Oct 22nd, 2021 - Patients are recruited from the attendee of the Infertility clinic and IVF unit in maternity hospital Ain shams university who are eligible for the trial. Procedure will be done by Prof . Dr Mostafa Fouad . and Lecturer Dr Ebtihal ElTaieb * Proper history taking, examination and investigations to: exclude other causes of infertility. Menstrual history: for assessment of oligo- or anovualtion. M...

Tissue Collection for Studies of Lymph Cancer
https://clinicaltrials.gov/ct2/show/NCT01676805

Oct 22nd, 2021 - Background: An estimated 79,190 people living in the United States will be diagnosed with lymphoma in 2012, including 9,060 cases of HL, 70,130 cases of non-Hodgkin s lymphoma (NHL) and multiple cases of adult T-cell leukemia/lymphoma. Laboratory investigations conducted in the Lymphoid Malignancies Branch, including analysis of cellular, molecular, genetic and genomic biology are attempting to...

Efficacy and Safety of Systemic Treatments of Bone Metastases From Kidney Cancer in Patients Treated With Targeted Therapies
https://clinicaltrials.gov/ct2/show/NCT03408652

Oct 22nd, 2021 - Multicenter, randomized, open-label, 2-arm, parallel-group, phase III study whose goal is to assess the efficacy and safety profile of bone-targeted treatments (Arm A: denosumab or zoledronic acid) versus the control arm (Arm B: no specific treatment) in patients with bone metastases under targeted therapy for Metastatic Renal Cell Carcinoma.

Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations
https://clinicaltrials.gov/ct2/show/NCT01861106

Oct 22nd, 2021 - Background: Genetic and sporadic mutations on one allele of the GATA2 gene lead to a syndrome termed MonoMAC. MonoMAC is characterized by: 1) infections with Mycobacterium avium complex (MAC) and other opportunistic infections, 2) deficiency of monocytes, B-lymphocytes, and Natural Killer (NK) cells in the peripheral blood, and 3) progression to myelodysplastic syndrome (MDS), chronic myelomono...

A Study of Pembrolizumab (MK-3475) in Combination With Belzutifan (MK-6482) and Lenvatinib (MK-7902), or Pembrolizumab/Quavonlimab (MK-1308A) in Combination With Lenvatinib, Versus Pembrolizumab an...
https://clinicaltrials.gov/ct2/show/NCT04736706

Oct 22nd, 2021 - The goal of this study is to evaluate the efficacy and safety of pembrolizumab plus belzutifan plus lenvatinib or pembrolizumab/quavonlimab plus lenvatinib versus pembrolizumab plus lenvatinib as first-line treatment in participants with advanced clear cell renal cell carcinoma (ccRCC). The primary hypotheses are (1) pembrolizumab plus belzutifan plus lenvatinib is superior to pembrolizumab plu...

Combination of Trametinib (MEK Inhibitor) and Hydroxychloroquine (HCQ) (Autophagy Inhibitor) in Patients With KRAS Mutation Refractory Bile Tract Carcinoma (BTC).
https://clinicaltrials.gov/ct2/show/NCT04566133

Oct 22nd, 2021 - Background: Among the new cases of bile tract carcinoma (BTC) that are diagnosed every year in the United States, there are approximately 6,500 cases of gallbladder carcinoma, 3,000 cases of extrahepatic cholangiocarcinoma, and 3,000 cases of intrahepatic cholangiocarcinoma. Current treatment options for patients with cholangiocarcinoma are limited and take no account of the known biological an...