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

Featured data showcase the transformative potential of HSC gene therapy to enable cross-correction and restore enzymatic ...

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.

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

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for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance.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

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 ...

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

Julien Grogg, PhD, MBA - CEO of Release Therapeutics Release Tx logo Dr. Julien Grogg will Lead the Company's Transition to Clinical ...

In January 2024, the company acquired Orchard Therapeutics Plc, which developed the world's most expensive drug for the treatment of a rare disease called metachromatic leukodystrophy, which is sold ...