Poor nutrition and lack of exercise lead to the increasing prevalence of obesity which, in turn, is the major predictor of diabetes and future risk of cardiovascular disease in western societies. v
Infants with the most severe form of spinal muscular atrophy (SMA) were more likely to show gains in motor function and were 47 percent more likely to survive without permanent assisted ventilation support when treated with a new medication, according to a study published today in the New England Journal of Medicine. The drug, nusinersen, performed so well that the study was stopped early and the treatment was approved shortly thereafter by the U.S. Food and Drug Administration (FDA) for all patients with this progressive neuromuscular disorder.
Celgene Corporation and bluebird bio, Inc. announced that bb2121, a chimeric antigen receptor T-cell (CAR-T) therapy targeting b-cell maturation antigen (BCMA) in previously treated patients with multiple myeloma, has been granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) and PRIority MEdicines (PRIME) eligibility by the European Medicines Agency (EMA).
Sangamo Therapeutics, Inc. (SGMO) today announced treatment of the first patient in the Phase 1/2 clinical trial ("the CHAMPIONS study") evaluating SB-913, an investigational in vivo genome editing therapy for people with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.
Their study found that with proper research methods and attention to privacy and ethical issues, social media big data can reveal important information concerning drug abuse, such as user-reported side effects, drug cravings, emotional states, and risky behaviors.
New data presented today showed that Jardiance® reduced the risk of cardiovascular death compared with placebo when added to standard of care in adults with type 2 diabetes and peripheral artery disease.
With fall well underway and winter just around the corner, chances are your doctor, pharmacist, and maybe even your mom are telling you it's time to roll up your sleeve for a flu shot. If you're inclined to ignore them, we get it: No one really enjoys being jabbed with a needle.
A man may soon be forever free of the previously incurable disease he was born with 44 years ago. On Monday, in a medical first, Brian Madeux received an experimental in-body gene-editing treatment intended to cure him of Mucopolysaccharidoses II, known as MPS II or Hunter syndrome, a rare disorder that causes progressive damage to the body's cells.
Keele University researchers have designed a new dye that can be used to observe the electrical activity of neurons in the brain and could lead to finding a new and more efficient way of treating neurological diseases, as presented at the Society for Neuroscience annual conference in Washington, D.C. this week.
Loxo Oncology struck a deal with Germany-based Bayer AG to develop and commercialize two cancer therapies in a deal that could be worth up to $1.55 billion for the Connecticut-based company.
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