A5010188573- Cystic Fibrosis Research Advances
- Hemoglobinopathies and Related Disorders
- Atrial Fibrillation Management and Outcomes
- Nematode management and characterization studies
- Antiplatelet Therapy and Cardiovascular Diseases
- Tracheal and airway disorders
- Advanced Breast Cancer Therapies
- HIV/AIDS drug development and treatment
- Cardiac electrophysiology and arrhythmias
- Iron Metabolism and Disorders
- Hepatitis C virus research
- Blood groups and transfusion
- Legume Nitrogen Fixing Symbiosis
- Prenatal Screening and Diagnostics
- HER2/EGFR in Cancer Research
- Renal Transplantation Outcomes and Treatments
- Blood Coagulation and Thrombosis Mechanisms
- Neonatal Respiratory Health Research
- Receptor Mechanisms and Signaling
- Parvovirus B19 Infection Studies
- Acute Myocardial Infarction Research
- Lung Cancer Research Studies
- Dialysis and Renal Disease Management
- Pharmacogenetics and Drug Metabolism
- Global Health Care Issues
Vertex Pharmaceuticals (United States)
2018-2025
Guangdong Academy of Medical Sciences
2025
Third Affiliated Hospital of Southern Medical University
2025
Guangdong Provincial People's Hospital
2025
Children’s Hospital at TriStar Centennial
2024
St. Jude Children's Research Hospital
2024
Morgan Stanley Children's Hospital
2024
Berlin Institute of Health at Charité - Universitätsmedizin Berlin
2018
Novartis (United States)
2016
Novartis (Switzerland)
2016
The inhibition of cyclin-dependent kinases 4 and 6 (CDK4/6) could potentially overcome or delay resistance to endocrine therapy in advanced breast cancer that is positive for hormone receptor (HR) negative human epidermal growth factor 2 (HER2).In this randomized, placebo-controlled, phase 3 trial, we evaluated the efficacy safety selective CDK4/6 inhibitor ribociclib combined with letrozole first-line treatment 668 postmenopausal women HR-positive, HER2-negative recurrent metastatic who had...
Cystic fibrosis is caused by mutations in the gene encoding cystic transmembrane conductance regulator (CFTR) protein, and nearly 90% of patients have at least one copy Phe508del CFTR mutation. In a phase 2 trial involving who were heterozygous for mutation minimal-function (Phe508del-minimal function genotype), next-generation corrector elexacaftor, combination with tezacaftor ivacaftor, improved clinical outcomes.We conducted 3, randomized, double-blind, placebo-controlled to confirm...
VX-445 is a next-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector designed to restore Phe508del CFTR protein function in patients with when administered tezacaftor and ivacaftor (VX-445–tezacaftor–ivacaftor).
The next-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector VX-659, in triple combination with tezacaftor and ivacaftor (VX-659–tezacaftor–ivacaftor), was developed to restore the function of Phe508del CFTR protein patients fibrosis.
Rationale: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be efficacious and safe in patients ≥12 years of age with cystic fibrosis at least one F508del-CFTR (cystic transmembrane conductance regulator) allele, but it has not been evaluated children <12 age. Objectives: To assess the safety, pharmacokinetics, efficacy ELX/TEZ/IVA 6 through 11 F508del-minimal function or F508del-F508del genotypes. Methods: In this 24-week open-label phase 3 study, (N = 66) weighing <30 kg...
Elexacaftor–tezacaftor–ivacaftor is a small-molecule cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen shown to be efficacious in patients with at least one Phe508del allele, which indicates that this combination can modulate single allele. In whose other CFTR allele contains gating or residual function mutation already effectively treated previous modulators (ivacaftor tezacaftor–ivacaftor), the potential for additional benefit from restoring protein unclear.
Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis by means of ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) at the erythroid-specific enhancer region
Phase 3 clinical trials showed elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was safe and efficacious in people with cystic fibrosis (CF) ≥1 F508del-CFTR allele. To assess long-term effects of ELX/TEZ/IVA under real-world conditions use, a 5-year observational registry-based study is being conducted. We report interim results from the first 2 years follow-up.The included CF US Cystic Fibrosis Foundation Patient Registry (CFFPR) who initiated between October 2019 December 2020. Pulmonary...
Background In two pivotal phase 3 trials, up to 24 weeks of treatment with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was efficacious and safe in patients cystic fibrosis (CF) ≥12 years age who have at least one F508del allele. The aim this study is assess long-term safety efficacy ELX/TEZ/IVA these patients. Methods 3, open-label, single-arm extension study, participants –minimal function (from a 24-week parent study; n=399) or – 4-week n=107) genotypes receive the same dose (ELX 200 mg...
Photoacoustic microscopy imaging could be used to evaluate the severity of liver fibrosis by means an analysis structural and functional characteristics lobules.
The hepatitis C virus protease inhibitor boceprevir is a strong of cytochrome P450 3A4 and 3A5 (CYP3A4/5). Cyclosporine tacrolimus are calcineurin immunosuppressants used to prevent organ rejection after liver transplantation; both substrates CYP3A4. This two-part pharmacokinetic interaction study evaluated with cyclosporine (part 1) 2). In part 1, 10 subjects received single-dose (100 mg) on day (800 3, concomitant cyclosporine/boceprevir 4. After washout, mg three times day) for 7 days...
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator gene (
Background. Boceprevir represents a new treatment option for hepatitis C (HCV)–infected patients, including those with HCV/human immunodeficiency virus coinfection; however, little is known about pharmacokinetic interactions between boceprevir and antiretroviral drugs. Methods. A randomized, open-label study to assess the ritonavir-boosted protease inhibitors (PI/r) was conducted in 39 healthy adults. Subjects received (800 mg, 3 times daily) 6 days then PI/r as follows: atazanavir (ATV) 300...
To analyze the pharmacokinetics of posaconazole administered as prophylaxis for invasive fungal infection (IFI) in neutropenic patients receiving chemotherapy acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS).Pharmacokinetic subanalysis a phase III, prospective, randomized, multicenter, evaluator-blinded trial comparing with standard azoles (fluconazole and itraconazole).One hundred ninety-four AML MDS who received oral suspension 200 mg 3 times/day meals nutritional...
1038 Background: Endocrine therapy (ET) is the basis of first-line (1L) treatment for HR+ ABC. However, ET resistance are almost universal. At first interim analysis (IA) MONALEESA-2 (NCT01958021), ribociclib (RIB; cyclin-dependent kinase 4/6 inhibitor) + letrozole (LET) significantly prolonged progression-free survival (PFS) vs placebo (PBO) LET in patients (pts) with HR+, HER2– 1 Here we report updated efficacy and safety data from a further ~11 months follow-up. Methods: Postmenopausal...
Oral temozolomide is approved in many countries for malignant glioma and melanoma some outside the USA. This study evaluated exposure equivalence safety of by intravenous infusion oral administration.Subjects with primary central nervous system malignancies (excluding lymphoma) received 200 mg/m(2) on days 1, 2 5. On 3 4, subjects 150 either as a 90-min one day or administration an alternate day.Ratio log-transformed means (intravenous:oral) area under concentration-time curve maximum...
This randomized, open-label, parallel-group study evaluated the effects of multiple-dose ketoconazole or rifampin on single- and pharmacokinetics vorapaxar. Healthy subjects randomly received one following three treatments (N = 12/group): (1) 400 mg once daily (QD) for 28 days (Days 1-28) single-dose vorapaxar 20 Day 7 followed by 2.5 QD 21 8-28); (2) 600 (3) placebo 8-28). Ketoconazole increased steady-state AUC(0-24 h) C(max) approximately twofold (GMR [90% CI]: 196% [173,222]; 193%...