The field of gene editing recently got a major boost from Massachusetts-based Beam Therapeutics, and the company’s Durham ...

Over 95% of babies now diagnosed with SMA are treated with Zolgensma gene therapy, per Daniel Grant, vice president for ...

Michelle Harlan, who has sickle beta thalassemia, wants to help others by getting a doctoral degree in clinical psychology.

Claire Berry lost her dad to the same devastating disease in 2000 when she was just 14 years-old, saying she knows how ...

Mutations in BRCA2—a gene known to repair damaged DNA and suppress tumor formation—can predict an individual's predisposition ...

Scientists have developed a new method that can grow motor neurons from skin cells, which may serve as a platform for ALS ...

Genprex (GNPX) announced that its research collaborators will present at the upcoming 2025 American Association for Cancer Research, or AACR, ...

Childhood-onset striatonigral neurodegeneration robs children of the ability to walk and talk by age five. Researchers at ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

Scientists have discovered more than a dozen genes that regulate the production of fetal hemoglobin in people of African ...

Nearly two-thirds of workplace bullies are bosses, according to the Workplace Bullying Institute. Here's what companies ...

Due to hemophilia A patients switching to newer treatments, Takeda Pharmaceuticals is discontinuing both Hemofil M and ...