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

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

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

The conclusions drawn by Zhang et al. may not align with current best practices and could risk creating false hope for MLD families. The Metachromatic Leukodystrophy Initiative (MLDi) critically ...

The distribution of diagnosed leukoencephalopathies was as follows: metachromatic leukodystrophy 62, X-linked adrenoleukodystrophy 60, Krabbe disease 39, Canavan disease 16, Alexander disease 13 ...

Among these innovations, if there is one name that is gaining a lot of attention recently in the medical field, it is precision medicine. Precision medicine at its core stands out as a revolutionary ...

Orchard Therapeutics to Receive WORLDSymposium™ New Treatment Award ...

specifically the lysosomal storage diseases such as metachromatic leukodystrophy, and Canavan disease, both of which result from mutations in enzymes in the sulphatide pathway. There are no cures ...

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therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance.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 ...

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 ...