$4.25 Million Gene Therapy Approved to Treat Rare Fatal Childhood Disorder, Allowing Normal Development

• A new $4.25 million gene therapy called Lenmeldy was approved to treat the rare and fatal genetic disorder metachromatic leukodystrophy (MLD) in children

• Lenmeldy repairs the cells of MLD patients so they can produce a missing enzyme needed to prevent nerve damage

• The therapy has allowed some children to now develop normally after over 12 years of follow-up

• The high price raises concerns about insurance coverage and access, but experts say the benefits justify the costs

• Earlier diagnosis through newborn screening could enable more children to benefit from the therapy before irreversible damage