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

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 ...

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

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for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance.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

The Minnesota Legislative Session was off to a rocky start in the House of Representatives and a historic power-sharing deal ...

won approval for the first gene therapy for children with a rare genetic disease called metachromatic leukodystrophy. Orchard priced the one-time treatment at $4.25 million, the most expensive ...

Libmeldy (atidarsagene autotemcel) was approved by the European Commission to treat metachromatic leukodystrophy (MLD) in December 2020, and subsequently rejected for NHS use by NICE last July on ...

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 ...

His team is now extending the X-ALD trial both within Europe and to the US, and a trial using the same strategy in metachromatic leukodystrophy is being planned by a team in Milan. Aubourg hopes ...

The study identifies conditions such as Metachromatic leukodystrophy (MLD) in babies. Further, it benefits hundreds of babies from earlier diagnosis and treatment that could help slow the ...

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 ...