Genetics

Experimental therapy may offer hope for rare genetic disorders

Researchers at Massachusetts General Hospital (MGH) have developed a new way to alleviate problems caused by dysfunctional mitochondria, which are the "powerhouses" that produce energy in cells. Their discovery, reported ...

Medical research

New treatment for muscular dystrophy wins US regulatory approval

Research led by Professor Steve Wilton and Professor Sue Fletcher and licensed to Sarepta Therapeutics has delivered a second treatment for Duchenne muscular dystrophy, with the drug gaining accelerated approval by the U.S. ...

Medical research

Low enrollment stymies completion of rare disease clinical trials

Rare diseases are anything but rare when taken together. In the United States alone, they affect nearly 30 million people. Yet clinical trials for rare diseases are frequently incomplete or go unpublished in the medical literature, ...

Medications

Scientists suggest new solution to the rare-disease problem

Thousands of rare diseases cumulatively affect millions of people across the globe, yet because each case is so rare doctors struggle to accurately diagnose and effectively treat individual patients. Every time a patient ...

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