Researchers have found that a new kind of gene therapy led to a dramatic decline in bleeding events in dogs with naturally occurring hemophilia A.
Researchers reported promising outcomes data for the first group of boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a fatal genetic immunodeficiency also known as "bubble boy" disease, who were treated as part of an international clinical study of a new form of gene therapy.
Triplets-and-more increasingly are the result of drugs given to women to make them produce eggs — not from using multiple embryos from IVF, or lab-dish fertilization, new research shows.
Researchers have identified a protein that causes loss of function in immune cells combatting HIV. The scientists report that the protein, Sprouty-2, is a promising target for future HIV drug development.
A government study offers a new theory on why the whooping cough vaccine doesn't seem to be working as well as expected. The research suggests that while the vaccine may keep people from getting sick, it doesn't prevent them from spreading whooping cough to others.
A novel immune-boosting drug combination eradicates glioblastoma brain cancer in mice, according to a new study.
Chemical engineers have developed a novel way to generate nanoparticles that can recognize specific molecules, opening up a new approach to building durable sensors for many different compounds.
A single overactive enzyme worsens the two core defects of diabetes—impaired insulin sensitivity and overproduction of glucose—suggesting that a drug targeting the enzyme could help correct both at once, according to new mouse studies.
A new nanotechnology-based technique for regulating blood sugar in diabetics may give patients the ability to release insulin painlessly using a small ultrasound device, allowing them to go days between injections.
After more than four years of investigation, researchers have discovered a promising treatment for osteoporosis, which is easily delivered in water soluble form.
The deadliest brain cancer requires grueling treatment with bleak prospects for survival. Now, researchers have discovered a key component to how these aggressive tumors grow that could lead to better solutions.
Princeton University officials decided Monday to make available a meningitis vaccine that hasn't been approved in the U.S. to stop the spread of the sometimes deadly disease on campus.
Researchers have developed a new technique for fighting deadly and hard-to-treat pancreatic cancer that uses two different types of nanoparticles, the first type clearing a path into tumor cells for the second, which delivers chemotherapy drugs.
The transplant anti-rejection drug rapamycin showed unexpected benefits in a mouse model of a fatal defect in the energy powerhouses of cells, the mitochondria.
Friendly microbes in the intestinal tracts (guts) of healthy American children have numerous antibiotic resistance genes, according to results of a pilot study by scientists.
The American Heart Association and American College of Cardiology have issued the first new guidelines in a decade for preventing heart attacks and strokes. Among other things, they call for twice as many Americans to consider taking cholesterol-lowering statin drugs.
Researchers studying how cancer spreads into bone have made a surprising discovery that suggests several investigational anti-cancer therapies just entering the drug-development pipeline may not have the desired effect.
U.S. experts are raising the alarm over the spread of drug-resistant malaria in several Southeast Asian countries, endangering major global gains in fighting the mosquito-borne disease that kills more than 600,000 people annually.
A team of scientists have identified a novel therapeutic approach for the most frequent genetic cause of ALS, a disorder of the regions of the brain and spinal cord that control voluntary muscle movement, and frontotemporal degeneration, the second most frequent dementia.
Researchers have launched an early-stage clinical trial of an investigational vaccine designed to prevent genital herpes disease.
Testosterone treatments may increase risks for heart attacks, strokes and death in older men with low hormone levels and other health problems, a big Veterans Affairs study suggests.
The generic anticonvulsant medication gabapentin shows promise as an effective treatment for alcohol dependence, based on the results of a 150-patient clinical trial of the medication. The study found that alcohol dependent patients using gabapentin were more likely to stop drinking or refrain from heavy drinking than those taking placebo.
An experimental vaccine to protect against respiratory syncytial virus (RSV), a leading cause of illness and hospitalization among very young children, elicited high levels of RSV-specific antibodies when tested in animals, according to a new report.
Cisplatin is a chemotherapy drug given to more than half of all cancer patients. The drug kills cells very effectively, but tumors can become resistant to the drug and grow back. A new study offers a possible way to overcome that resistance.
A bone drug already on the market for osteoporosis may kill chronic myelogenous leukemia (CML) stem cells thought to persist in the bone marrow after standard therapy, lowering the likelihood of disease recurrence, according to a new study.