Healio

FDA approves Lenmeldy for early juvenile metachromatic leukodystrophy

Please provide your email address to receive an email when new articles are posted on . Metachromatic leukodystrophy is an incurable rare genetic disease. At 5 years of age, 71% of children given ...
Medscape

FDA OKs First Gene Therapy for Metachromatic Leukodystrophy

The US Food and Drug Administration (FDA) has approved the first gene therapy for metachromatic leukodystrophy (MLD), a rare and debilitating genetic disease affecting the brain and nervous system.
Washington Times

New FDA-approved gene therapy for children will cost record $4.25 million

A new one-time gene therapy that treats a rare disease in children will cost an eye-popping $4.25 million, making it the world’s most expensive drug. The Food and Drug Administration approved Lenmeldy ...
Nature

Effects of atidarsagene autotemcel gene therapy on peripheral nerves in late-infantile metachromatic leukodystrophy

The Nature Index 2025 Research Leaders — previously known as Annual Tables — reveal the leading institutions and countries/territories in the natural and health sciences, according to their output in ...
Monthly Prescribing Reference

Gene Therapy Candidate Gets Priority Review for Metachromatic Leukodystrophy

Credit: Shutterstock. OTL-200 is an ex vivo autologous hematopoietic stem cell gene therapy. The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application ...