Grammy-nominated Hozier is known for the hit song Take Me To Church released in 2013. His 2014 a self-titled album made it to ...

Passage Bio is outsourcing future analytical testing services in a cost-cutting move that will further cut its workforce, a ...

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

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

The first baby to have a cord blood sample taken as part of the study at Jessop Wing was Sophia Bird, who was born on December 16, 2024. In a joint statement, Sophia’s mum Alice and dad Robert said: ...

vumc.nl Objective To determine whether proton magnetic resonance spectroscopic imaging is useful in predicting clinical course of patients with metachromatic leukodystrophy (MLD), an inherited white ...

brittle bone disease as well as lesser-known disorders such as metachromatic leukodystrophy (MLD) in hundreds of babies shortly after birth rather than when symptoms might appear later in childhood.

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 ...

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 times in ...

The central region of the country exhibited the highest prevalence of LDs (48.5%). The most common LD was metachromatic leukodystrophy (MLD), and it accounted for 25.3%. The most common disorder based ...

POLR3-related or 4H (hypomyelination, hypodontia, and hypogonadotropic hypogonadism) leukodystrophy is one of the most common types of hypomyelinating leukodystrophy for which no curative treatment or ...