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"FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy by Orchard"

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Source: FDA.gov
"FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy by Orchard"
Photo: FDA.gov
TL;DR Summary

The FDA has approved Lenmeldy, the first gene therapy for children with metachromatic leukodystrophy (MLD), a rare genetic disease affecting the brain and nervous system. Lenmeldy is a one-time, individualized single-dose infusion made from the patient’s own stem cells, genetically modified to include functional copies of the ARSA gene. Clinical trials showed that treatment with Lenmeldy significantly reduced the risk of severe motor impairment or death compared with untreated children. The most common side effects are fever and low white blood cell count, and patients should be monitored for potential complications.

Topics:business#fda#gene-therapy#health#lenmeldy#metachromatic-leukodystrophy#rare-diseases
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  1. FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy  FDA.gov
  2. Orchard's therapy for neuron disease in children wins FDA approval  STAT
  3. After Kyowa Kirin buyout, Orchard scores FDA approval for first MLD gene therapy in the US  FiercePharma
  4. Recon: FDA approves Orchard's gene therapy Lenmeldy; Pfizer sells partial stake in Haleon  Regulatory Focus
  5. First gene therapy to treat children with metachromatic leukodystrophy approved by FDA  Contemporary Pediatrics

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