17 May 2017 - In September, the FDA approved a controversial drug to treat Duchenne muscular dystrophy, a rare but deadly disease that affects boys. 

That was a reversal from six months earlier, when an FDA advisory committee had recommended against the drug’s approval and said it was not effective, Forbes reported. 

What tipped the scales in favour of the Exondys 51 drug? The voices of parents and children with the disease.

Read Forbes article