Safety and efficacy profile of first-in-class oral p53 reactivator rezatapopt
1. In this phase 1-2 single-group trial, the p53 reactivator rezatapopt was well-tolerated in adults with solid tumors harboring the...
Ex vivo gene therapy for cystinosis had acceptable safety and improved disease biomarkers
1. In this phase 1–2 open-label study, autologous lentiviral hematopoietic stem-cell gene therapy for cystinosis was associated with sustained engraftment...
Safety and efficacy of novel antibody-oligonucleotide therapy for myotonic dystrophy type 1
1. In this phase 1-2 randomized controlled trial involving adults with myotonic dystrophy type 1, del-desiran was well-tolerated in most...
Oral nonstatin medication significantly lowers cholesterol in high-risk patients
1. In this randomized controlled trial, once-daily oral enlicitide was associated with sustained reductions in low-density lipoprotein cholesterol levels compared...
Chimeric antigen receptor T-cell therapy may induce sustained remission in multirefractory autoimmune hemolytic anemia
1. In this prospective cohort study, CD19-directed chimeric antigen receptor (CAR) T-cell therapy was found to elicit a complete response...
![siRNA against antithrombin alleviates symptoms of hemophilia [PreClinical]](https://www.2minutemedicine.com/wp-content/uploads/2015/04/clot-CCWiki-350x250.jpg)
Tenecteplase not associated with visual improvement in central retinal artery occlusion
1. In this randomized controlled trial of adults with central retinal artery occlusion, tenecteplase administration shortly after symptom onset did...
Palbociclib as adjunct maintenance therapy prolongs progression-free survival in some types of metastatic breast cancer
1. This randomized controlled trial found that, in patients with hormone-receptor-positive, HER2-positive metastatic breast cancer, the addition of palbociclib to...
Sacituzumab govitecan plus pembrolizumab improves survival in advanced triple-negative breast cancer
1. Among patients with previously untreated programmed death-ligand 1 positive, triple-negative breast cancer, first-line sacituzumab govitecan plus pembrolizumab significantly prolonged...
Fremanezumab reduces migraine frequency in children and adolescents with episodic migraine
1. In this randomized controlled trial involving pediatric patients, fremanezumab led to greater reductions in the number of migraine days...
Brain-penetrant enzyme replacement therapy improves biomarkers in mucopolysaccharidosis type II
1. In this open-label phase 1–2 study, pediatric patients with mucopolysaccharidosis type II treated with tividenofusp alfa showed substantial reductions...
