muscular dystrophy

Gene therapy has helped a 9-year-old boy regain enough muscle strength to run. If successful in others, the treatment could change the lives of thousands of children with Duchenne muscular dystrophy.

Many Duchenne muscular dystrophy patients in the U.S. have imported a medicine called deflazacort for about $1,200 a year. A brand-name version just approved for sale in America costs $89,000.

A voucher awarded to a company that finds a treatment for a rare childhood disease can be sold to the highest bidder and then used to fast-track the review of another drug. Are the prizes worth it?

The Food and Drug Administration approved a muscular dystrophy drug despite deeply flawed evidence. Was the decision a dangerous precedent or flexible pragmatism reflecting patients' values?

A drug being tested against Ebola makes use of new scientific insights that could prove useful for treating other illnesses, including one that is inherited.