Julianna Archibald sits with her siblings Josiah Archibald, Mason Archibald, and Elijah Phillips, all three of whom have Barth syndrome. Josanne Archibald/Handout via REUTERS
Today's recommended read is an exclusive from my colleague Patrick Wingrove. He reports on the US FDA approving a pricey rare disease drug despite findings by its data reviewers that the treatment, while safe, was no more effective than a placebo.
The Food and Drug Administration on September 19 gave its backing to Stealth Biotherapeutics' elamipretide, which will be sold as Forzinity and priced at up to nearly $800,000 a year, although FDA documents show nearly a dozen reviewers recommended against approval.
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