12/5/2023 0 Comments Bubble boy diseaseThe treatment is a type of gene therapy: Doctors will give Seersha a normal copy of the defective gene that disabled Seersha’s immune system. Seersha Sulack's parents learned their daughter had it when she was five days old. In the US, about eight babies a year are born with ADA-SCID. Donald Kohn, a UCLA scientist who has been working on the treatment for nearly 40 years. Currently, 26 children in the US and Canada are on the waiting list to get the therapy, according to Dr. The treatment is for a particular type of severe combined immunodeficiency called ADA-SCID that’s extremely rare – in the US, eight babies a year are born with it. She’ll become only the second child in the US in the past five years to receive it the first child received it earlier this month. Seersha is expected to get the treatment next month. “You have an effective therapy like (this one) and people can’t get access to it.” Brooks, acting director of the Division of Rare Diseases Research Innovation at the National Institute of Health’s National Center for Advancing Translational Sciences. “It’s a pretty tough situation,” said Dr. The treatment she’s been waiting for had stunning, near-perfect results in a clinical trial, but it’s been sitting on the shelf for years in the US because the pharmaceutical company that once owned the license abandoned it when it decided not to not to pursue approval from the US Food and Drug Administration. Later this spring, a little girl in California who essentially has no immune system will receive a lifesaving treatment for “bubble boy disease” thanks to the persistence of a dogged group of parents, a pediatrician, a veteran newsman and a few episodes of “Grey’s Anatomy.”įive-year-old Seersha Sulack has the same rare disease portrayed in the 1976 John Travolta movie, “ The Boy in the Plastic Bubble.” A germ – even a common cold – could kill her, and so she stays away from anyone outside her immediate family.
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