Finally, it seemed, Sarah Jenssen would have a chance to get a treatment that her family hoped could help her.
A gene therapy for Sarah’s condition, Duchenne muscular dystrophy, won a wide approval from the Food and Drug Administration earlier this year. The authorization encompassed most patients with the degenerative neuromuscular disorder, regardless of their age or whether or not their disease had advanced to the point that they used wheelchairs.
But last month, the Jenssens’ insurer denied Sarah coverage for the therapy, which has a list price of $3.2 million. The insurer said it would only cover the treatment, Sarepta Therapeutics’ Elevidys, for patients who were still able to walk on their own, putting it at odds with the FDA’s label. Though she could still use the bathroom independently and dress herself, Sarah, 15, had started relying on a wheelchair.
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