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

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

Among these innovations, if there is one name that is gaining a lot of attention recently in the medical field, it is ...

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

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.

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for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance.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

After Paul Jasman died from a rare disease last summer, his father began donating medical items to a Fredericksburg program ...

This video features insights from leading experts on metachromatic leukodystrophy (MLD), a rare and progressive ...

After Paul Jasman died from a rare disease last summer, his father began donating medical items to a Fredericksburg program ...