For Wedam Minyila, a 19-year-old with sickle cell disease, a recent hospital visit marked a potential turning point. He was among the first patients worldwide to undergo commercial treatment for the genetic condition, which could lead to a cure. Though still in its early stages, the gene therapy holds immense promise for the over 100,000 people in the U.S. living with sickle cell disease, offering the possibility of a life free from excruciating pain and debilitating complications.
WASHINGTON — For Wedam Minyila, hospital rooms have always meant blinding pain. “Like someone is jamming a knife in me,” he said. But for a brief moment on a recent December morning, Wedam, 19, who has sickle cell disease, allowed himself to believe what his doctors had been telling him for months: This visit could be the first step to a cure.
” “Remember, this is a very high-risk treatment requiring high-dose chemotherapy with potential for complications,” he said. “So it’s not something that we would want to ramp up too rapidly.” The monthslong treatment Casgevy from Vertex Pharmaceuticals comes with a list price of $2.3 million. Bluebird Bio’s Lyfgenia is listed at $3.1 million. Neither includes the cost of care to stay in the hospital or for chemotherapy.
GENE THERAPY SICKLE CELL DISEASE MEDICAL BREAKTHROUGH CRISPR TREATMENT
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