Throughout the course of multiple sclerosis, gradually progressive neurologic impairment can occur, which has been called disability accrual. Current disease-modifying therapies for multiple sclero…
In this editorial, the authors describe the foundations of an N-of-1 gene-editing study to treat an infant with a urea-cycle disorder.
Base editors can correct disease-causing genetic variants. After a neonate had received a diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency, a disease with an estimated…
In this editorial, the authors describe the foundations of an N-of-1 gene-editing study to treat an infant with a urea-cycle disorder.
In this editorial, the authors describe the foundations of an N-of-1 gene-editing study to treat an infant with a urea-cycle disorder.
Combined treatment with a tyrosine kinase inhibitor and blinatumomab has converted Ph-positive ALL, once the most deadly hematologic cancer, to a disease with long-term survival…
Combined treatment with a tyrosine kinase inhibitor and blinatumomab has converted Ph-positive ALL, once the most deadly hematologic cancer, to a disease with long-term survival…
Combined treatment with a tyrosine kinase inhibitor and blinatumomab has converted Ph-positive ALL, once the most deadly hematologic cancer, to a disease with long-term survival…
Quick Take Video Summary from The New England Journal of Medicine — Antiretroviral Therapy for Children with HIV in Africa
Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Nexiguran ziclumeran (nex-z) is an investigational therapy based on CRISPR-Cas9 (clustered regularly i…
Telangiectasias, erythrocytosis, monoclonal gammopathy, perinephric-fluid collection, and intrapulmonary shunting are characteristic of the TEMPI syndrome. A case report documents improvement with …
