She’s doing so well now and can do everything a normal child her age would do.”
#Scid gene therapy skin
Six months after she was given the gene therapy her skin was clean and healthy and the other symptoms got a lot better. “Before, her nappy rash was always bleeding and sore, she was being sick and losing weight. Sarah was referred to GOSH for gene therapy and several years after her treatment is doing very well: It was horrible to not be able to do normal things with my own daughter.” Everything had to be highly sterilised to keep her safe. I saw it was SCID and I was incredibly upset… I wasn’t allowed to kiss my daughter or sleep next to her. “I remember the exact time I saw the paper that had the blood test results on and the diagnosis. She was less than ten days old when her mum Maria became concerned about her daughter’s weight loss and sore, bleeding nappy rash that wouldn’t heal. One of the patients treated was four-year-old Sarah from South Yorkshire. In the two cases in which treatment wasn’t successful, both children were able to return to current standard treatments, with one eventually receiving a bone marrow transplant.No serious side effects have so far been reported, with generally mild or moderate complications experienced from the necessary preparation for the gene therapy. Of these, 48 are no longer showing symptoms of ADA-SCID, although they will have lifelong monitoring. Two to three years after the treatment, all of the 50 children treated with the new gene therapy at Great Ormond Street Hospital, UCLA Mattel Children’s Hospital and the National Institutes of Health (NIH) are alive and well. If approved, gene therapy would be a welcome new treatment option for ADA-SCID as it is a one-time procedure that has the potential to provide life-long results. These treatments are expensive and therefore out of reach for patients in many countries. If a matched bone marrow donor is not available, patients require lifelong ADA injections along with preventative medicines. The standard treatment for ADA-SCID involves once or twice weekly injections of the ADA enzyme until a matched bone marrow donor – usually a close family member – can be found. In a new study published in the New England Journal of Medicine, co-lead authors Professor Donald Kohn (UCLA) and Dr Claire Booth (GOSH) reported the two- to three-year outcomes of 50 children who were treated in clinical trials with an experimental stem cell gene therapy for ADA-SCID between 20.
Gene therapy offers a potential cure to children born without an immune system.Staying safe at GOSH and outside the hospitalĬoming to GOSH for a day or inpatient admissionĬoming to GOSH for an outpatient appointment Our new visiting guideline after COVID-19
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