Muscular dystrophy, which affects approximately 250,000 people in the U.S., occurs when damaged muscle tissue is replaced with fibrous, fatty or bony tissue and loses function. For years, scientists have searched for a way to successfully treat the most common form of the disease, Duchenne Muscular Dystrophy (DMD), which primarily affects boys.

An FDA-approved drug for leukemia improved cognition, motor skills and non-motor function in patients with Parkinson's disease and Lewy body dementia in a small phase I clinical trial, report researchers at Georgetown University Medical Center (GUMC) in Washington. In addition, the drug, nilotinib (Tasigna® by Novartis), led to statistically significant and encouraging changes in toxic proteins linked to disease progression (biomarkers).

Drugs produced using biotechnology are among the most expensive medicines available. Biosimilars that offer a real alternative are becoming increasingly available on the market. The promise of biosimilars is that, once patent protection has elapsed for established biologics, more patients will be able to access effective and well-tolerated drugs.

Researchers at University of California, San Diego School of Medicine and Skaggs School of Pharmacy and Pharmaceutical Sciences have found that the breast cancer drug tamoxifen gives white blood cells a boost, better enabling them to respond to, ensnare and kill bacteria in laboratory experiments. Tamoxifen treatment in mice also enhances clearance of the antibiotic-resistant bacterial pathogen MRSA and reduces mortality.

A new study finds that rising placebo responses may play a part in the increasingly high failure rate for clinical trials of drugs designed to control chronic pain caused by nerve damage. Surprisingly, however, the analysis of clinical trials conducted since 1990 found that the increase in placebo responses occurred only in trials conducted wholly in the U.S.; trials conducted in Europe or Asia showed no changes in placebo responses over that period.

Cancer specialists have long looked at genetic mutations and DNA copy changes to help predict patient survival and drug sensitivity. A study led by The University of Texas MD Anderson Cancer Center has opened up yet another avenue for understanding the biological reasons why some people live longer or respond better to treatment - RNA editing events.

A pair of studies by researchers at the University of Massachusetts Medical School, the University of Trento in Italy, and the University of Geneva in Switzerland, point to a promising new anti-retroviral strategy for combating HIV-1. The two studies, published online in Nature, show that the host cell membrane proteins SERINC5 and SERINC3 greatly reduce the virulence of HIV-1 by blocking the ability of the virus to infect new cells.