In the pipeline: Drugs for a devastating condition
WHITNEY WELDON IS ONE OF ABOUT 800 PEOPLE IN THE WORLD WITH A RARE DISEASE KNOWN AS FOP. (ALICE PROUJANSKY FOR STAT)
For decades, the rare disease known as fibrodysplasia ossificans progressiva, or FOP, was a medical curiosity, a condition without a treatment or even a biological explanation. A small community of patients simply adjusted to grim realities: Their tissue would turn to bone, their joints would freeze, and their life expectancy would be short.
Now, however, after decades of work by researchers, three companies may be on the brink of leaping a “tough scientific hurdle,” as one CEO put it, and bringing treatments to market. The most advanced drug could win Food and Drug Administration approval next year.
STAT’s Damian Garde has the story of a patient group, some dedicated researchers, and a fair amount of scientific serendipity.
Read more.
Now, however, after decades of work by researchers, three companies may be on the brink of leaping a “tough scientific hurdle,” as one CEO put it, and bringing treatments to market. The most advanced drug could win Food and Drug Administration approval next year.
STAT’s Damian Garde has the story of a patient group, some dedicated researchers, and a fair amount of scientific serendipity.
Read more.
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