Metachromatic leukodystrophy is a genetic condition that occurs due to an enzyme deficiency. Patients with this disorder have ...

The Metachromatic Leukodystrophy Initiative (MLDi) critically responds to the findings of Zhang et al. on lentivirus-modified ...

Orchard Therapeutics has revealed its US pricing plans for Lenmeldy, its gene therapy for metachromatic leukodystrophy (MLD), placing a $4.25 million price tag on the one-shot treatment.

Khloe, from Nashville, was diagnosed last year with metachromatic leukodystrophy, a rare genetic disorder that attacks the ...

Orchard Therapeutics has tasked Robin Kenselaar with accelerating growth of Lenmeldy, the world’s most expensive drug, ...

Orchard Therapeutics' gene therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance. The agency said that while there ...

And yet everything is different. July 2024 is when Jasman passed away from a rare condition known as Metachromatic leukodystrophy, often abbreviated as MLD. “MLD causes the brain to separate ...

Featured data showcase the transformative potential of HSC gene therapy to enable cross-correction and restore enzymatic function in a variety of tissues and organs, including the CNSTOKYO, LONDON and ...

untreated sibling pairs with early-onset metachromatic leukodystrophy* Date/Time: Friday, February 7 at 2:00 p.m. Presenter: Karen Bean Details of the poster presentations are as follows (all ...