This rare genetic disease in children has a treatment. It costs $4.25 million
- Updated Thursday Mar 21 2024
This rare genetic disease in children has a treatment. It costs $4.25 million
"The FDA approval of Lenmeldy opens up tremendous new possibilities for children in the US with early-onset MLD who previously had no treatment options beyond supportive and end-of-life care,” said Bobby Gaspar, co-founder and chief executive officer of Orchard Therapeutics.
As per Gasper, MLD is a rapidly progressing, life-limiting and ultimately fatal rare disease that has a devastating impact on afflicted children and their families.
"This achievement is the culmination of decades of research and development in partnership with our academic and clinical collaborators at the San Raffaele-Telethon Institute for Gene Therapy," said Gasper.
Lenmeldy costs at $4.25 million for one-time treatment.
WHAT IS MLD?
MLD or metachromatic leukodystrophy is a progressive, highly rare, genetic neurometabolic disease that affects the nervous system. It causes a lack of a vital enzyme, leading to the build-up of harmful substances in the brain and nerves.
Symptoms include developmental delays, muscle weakness, and loss of skills. MLD progresses rapidly and can be fatal.
Lenmeldy has the potential to "stop or slow the progression" of the disease with a single treatment, especially when it is administered to the child at the start of the symptoms.
Share News
Please Login to place a comment
Login Now
-
LATEST
-
POPULAR
