A5030103394- Complement system in diseases
- Blood groups and transfusion
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Genetic Syndromes and Imprinting
- Hemoglobinopathies and Related Disorders
- Liver Disease and Transplantation
- Poisoning and overdose treatments
- Renal Diseases and Glomerulopathies
- Pesticide Exposure and Toxicity
- Liver Disease Diagnosis and Treatment
- Erythropoietin and Anemia Treatment
- Neuroendocrine Tumor Research Advances
- Occupational Health and Safety Research
- Adenosine and Purinergic Signaling
- Cardiovascular and exercise physiology
- Heart Failure Treatment and Management
- Anesthesia and Neurotoxicity Research
- Congenital Ear and Nasal Anomalies
- Takotsubo Cardiomyopathy and Associated Phenomena
- Electroconvulsive Therapy Studies
- Cardiac Fibrosis and Remodeling
- Liver Diseases and Immunity
- Multiple Sclerosis Research Studies
- Congenital heart defects research
- Neonatal and fetal brain pathology
Apellis Pharmaceuticals (United States)
2021-2024
National Center for Environmental Health
2023
Centers for Disease Control and Prevention
2023
Emory University
2020-2022
Washington Poison Center
2022
Biogen (United States)
2015
University of California, Davis
2007
Presbyterian Hospital
1997
Casa Sollievo della Sofferenza
1997
Albert Einstein College of Medicine
1996-1997
Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by complement-mediated intravascular hemolysis leading to anemia, fatigue, and potentially life-threatening thrombotic complications. Breakthrough (BTH) was first described in patients with PNH treated terminal complement C5 inhibitors when reoccurred despite treatment. Pegcetacoplan, the proximal C3 inhibitor, offers broad control PNH. While experience of managing BTH on documented, very limited guidance exists for inhibitors. This...
Abstract Sox6, a member of the Sox family transcription factors, is highly expressed in skeletal muscle. Despite its abundant expression, role Sox6 muscle development not well understood. We hypothesize that, fetal muscle, functions as repressor slow fiber type‐specific genes. In wild‐type mouse, differentiation fast and fibers becomes apparent during late stages (after approximately embryonic day 16). However, null‐ p 100H mutant all maintain characteristics, evidenced by expression myosin...
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening disease with symptoms of hemolysis thrombosis. Current therapies for this complement-mediated rely predominantly on inhibition the C5 complement protein. However, data treatment responses quality life in C5-inhibitor (C5i)-treated PNH patients are scarce. The objective study was to determine C5i effects clinical parameters, symptoms, life, resource use patients. This cross-sectional surveyed 122 individuals USA...
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening disease characterized by complement-mediated hemolysis and thrombosis. Pegcetacoplan, the first targeted complement component 3 (C3) PNH therapy, was safe efficacious in treatment-naive pre-treated patients with five clinical trials. The 307 open-label extension (OLE) study (NCT03531255) non-randomized, multicenter of long-term safety efficacy pegcetacoplan adult who completed parent study. All received pegcetacoplan....
Background Long-term β-adrenergic blockade does not appear to be associated with drug-induced training in patients congestive heart failure (CHF); whether exercise can increase peak aerobic capacity CHF who are treated blockers is currently unknown. Methods and Results We studied 23 were carvedilol or propranolol addition ACE inhibitors, furosemide, digoxin. Of the carvedilol, 8 underwent remained sedentary. All 7 training. Peak oxygen consumption (mL·kg −1 ·min ) was serially measured...
Paroxysmal nocturnal hemoglobinuria (PNH), a rare acquired hematologic disorder, can be treated with C5 inhibitors (C5i) such as eculizumab or ravulizumab. This retrospective study is the first to describe real-world treatment patterns and changes in PNH-monitoring laboratory tests among C5i-treated US patients. Data were extracted from TriNetX Dataworks Network included patients PNH diagnosis between 1 January 2010, 20 August 2021. Patients stratified into three cohorts based on their C5i...
Multiple sclerosis (MS) is a debilitating disease that negatively impacts patients' lives.ENABLE assessed the effect of long-term prolonged-release (PR) fampridine (dalfampridine extended release in United States) treatment on patient-perceived health impact patients with MS walking impairment.ENABLE was 48-week, open-label, Phase 4 study PR-fampridine 10 mg twice daily. Patients who showed any improvement Timed 25-Foot Walk speed at weeks 2 and 12-item Walking Scale score week remained...
In the absence of head-to-head trials, this study compared treatment outcomes with C3 complement inhibitor pegcetacoplan versus C5 eculizumab or ravulizumab in inhibitor-naïve patients paroxysmal nocturnal hemoglobinuria (PNH). A matching-adjusted indirect comparison was conducted using individual patient data from arm PRINCE trial (NCT04085601; n = 34) and aggregate (n 125) 121) arms ALXN1210-PNH-301 (NCT03056040). Clinical quality life endpoints were evaluated after matching two trials on...
We determined normalization rates for hemoglobin, lactate dehydrogenase (LDH), and fatigue in patients with paroxysmal nocturnal hemoglobinuria (PNH) treated pegcetacoplan (PEG) the PEGASUS (NCT03500549) PRINCE (NCT04085601) phase III trials. Enrolled had PNH hemoglobin < 10.5 g/dL despite ≥ 3 months of eculizumab (ECU) [PEGASUS], or were complement component 5 (C5) inhibitor-naive, receiving supportive care only, less than lower limits normal (LLN) [PRINCE]. Hematologic endpoints greater...
Abstract Prader-Willi syndrome (PWS) is a complex developmental genetic disorder associated with hypotonia, poor feeding in neonates, onset of hyperphagia early childhood, and shorter overall life expectancy. Prior epidemiology studies PWS have examined smaller populations, limited research US population. The aim this study was to provide contemporary estimate prevalence annual all-cause mortality the using large administrative medical claims dataset. Methods: patients were identified...
Summary Protein S is a nonenzymatic and vitamin K‐dependent cofactor of activated protein C. Without S, the anticoagulant function C almost depleted thrombotic events occur. We report parturient with hereditary deficiency in whom risk thromboembolism was further complicated by pregnancy who required emergency Caesarean section for fetal distress.
Abstract Prader-Willi syndrome (PWS) is a rare, complex neuro-developmental genetic disorder characterized by hyperphagia and abnormal food-related behaviors that contribute to severe morbidity early mortality significant burden on patients caregivers. While majority of people with PWS obese, observed in both obese non-obese PWS. There currently no approved treatment for People have increased circulating levels the orexigenic hormone acylated ghrelin (AG) relative deficit unacylated (UAG),...
Lacrimators are used by individuals for personal defense and police crowd control during periods of civil unrest. Increased public awareness about their use has raised concerns application safety.
The Veratrum genus is composed of plants containing a diverse set steroidal alkaloids. plant material has been utilized for centuries as herbal medicines, however the alkaloids have such low therapeutic index that they are not used in modern medicine. Here we report an incident inadvertent ingestion V. parviflorum by hikers Georgia allowed detection, and several instances identification from plant, correlated their presence within patient blood breast milk specimens.Eight patients, three...