Jennifer Hill Blair experienced a mix of hope and despair when she learned her son Keise could finally receive a life-changing treatment for Duchenne muscular dystrophy at Cincinnati Children’s Hospital. However, just as approval from the insurance company came on July 9, Keise’s birthday, the treatment’s availability was jeopardized by the FDA’s decision to withdraw it from the market due to safety concerns.
This left many families, primarily of young boys with Duchenne, devastated as appointments were postponed indefinitely. The situation was exacerbated by rapidly changing news updates, causing emotional turmoil for affected families. Experts emphasized the profound impact on the children and their families, highlighting the unfairness and stress of the ongoing uncertainty. The once-promising gene therapy, designed to slow the progression of this fatal condition, now faces a complicated path back to the market amid ongoing regulatory challenges.
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