A drug company abandoned a treatment for  ‘bubble boy disease.’ After a 5-year fight, this little California girl is about to get it - News Summed Up

A drug company abandoned a treatment for  ‘bubble boy disease.’ After a 5-year fight, this little California girl is about to get it


The Sulacks weighed their options: Have a transplant with a match that was less than ideal — far less — or wait for gene therapy to become available. The news release didn’t say anything else about the SCID gene therapy. Or was the company abandoning its plans for SCID gene therapy altogether? In February, 2021, the parents of more than 20 children who were waiting for the gene therapy treatment, including the Sulacks, wrote a letter to Gaspar. Insurance companies have sometimes balked at paying for gene therapy, which is typically given in one treatment.


Source: CNN April 27, 2023 15:45 UTC



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