A toddler in Minnesota is walking after being given an expensive gene therapy to treat her spinal muscular atrophy, Newsweek reports.
According to KARE in Minneapolis, Maddy Smith's parents fought for her to have the treatment. The US Food and Drug Administration approved onasemnogene abeparvovec, (Novartis' Zolgensma) for children under the age of two shown by genetic testing to have one of three forms of SMA, which causes paralysis, breathing difficulties, and death. But the treatment is expensive with a list price of $2.1 million. KARE says Maddy's parents had to convince the State of Minnesota, through which Maddy was insured, to cover the test before she turned 2 years old. Minnesota, it reports, initially denied the request, but then allowed the treatment, which Maddy received a day before her birthday.
But now, KARE reports Maddy has begun to walk. "To see someone with improvement, such dramatic improvement, essential normal milestones is certainly very impressive," her doctor the University of Minnesota's Peter Karachunski tells it.