A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...

Faulty versions of the LMNA gene can cause a wide range of health problems, including heart muscle disease (dilated ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the ...

MIT scientists have found a way to make gene editing far safer and more accurate — a breakthrough that could reshape how we ...

Parkinson’s disease currently affects nearly one million Americans with no specific known cause and no cure. Treatments have ...

Medication has long been the cornerstone of treatment for people with epilepsy, but it doesn't stop seizures for everyone and ...

From easing tremors to opening the blood-brain barrier, focused ultrasound is emerging as one of medicine’s most promising ...

Nine-year-old Andy Cash was diagnosed with Adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID), a rare ...

Life expectancy has soared and interest in the disease is growing, but breakthrough gene therapies are extraordinarily ...