Stanford Medicine pediatric hematologist Agnieszka Czechowicz, MD, Ph.D., has devoted her research career to improving ...

Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers. The intermediate ...

Blood clotting disorders can be inherited or acquired and lead to either excessive bleeding or too much clotting. Hypercoagulable disorders can cause dangerous blockages due to excessive blood ...

Vertex Pharmaceuticals is hoping to build upon its existing rare blood disease treatment portfolio, teaming up with Orna Therapeutics’ ReNAgade in efforts to develop next-gen gene therapies for sickle ...

A blood test is a medical procedure where a sample of a person's blood is taken (called a blood draw) and sent to a lab for analysis. Knowing the results of blood tests helps a physician better ...

A certain blood protein regarded as an early indicator of Alzheimer's disease also appears to play a role in other disorders.

A certain blood protein regarded as an early indicator of Alzheimer's disease also appears to play a role in other disorders.

In Q1 2025, the Company will discuss the plans for NDA submission under a potential accelerated approval path for bitopertin in erythropoietic protoporphyria (EPP), following a Type C meeting with FDA ...

Scientists develop a blood test that detects Alzheimer’s by measuring protein structural changes rather than protein quantity.