Time is running out for kids with a rare disease, parents say, unless the FDA acts
The drug Elamipretide from Stealth BioTherapeutics treats an ultrarare, fatal disease called Barth syndrome. Small studies say it's safe and effective. But the FDA denied approval, leaving children with few options.
The drug Elamipretide from Stealth BioTherapeutics treats an ultrarare, fatal disease called Barth syndrome. Small studies say it's safe and effective. But the FDA denied approval, leaving children with few options.
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