Tag: Metachromatic leukodystrophy

With a list price of $4.25 million per dose, Lenmeldy became the most expensive prescription drug on the market after its FDA approval in early 2024. This staggering figure highlights how recent breakthroughs in gene therapy are redefining what is the most expensive prescription drug and sparking critical discussions about access and cost.

Metachromatic leukodystrophy (MLD) is a rare inherited disease, and for eligible patients, the gene therapy Libmeldy offers a potentially curative, one-time treatment. The process to administer this revolutionary therapy is complex, involving several meticulous steps from initial cell collection to final intravenous infusion at a qualified treatment center.

Affecting approximately one in every 100,000 live births, metachromatic leukodystrophy (MLD) is a devastating genetic disorder. For eligible patients, the innovative one-time gene therapy, Libmeldy, offers a new approach to treatment, but understanding **how does libmeldy work** requires a deep dive into advanced molecular science.