SSIEM 2024
SSIEM is the Society for the Study of Inborn Errors of Metabolism Scientists, clinicians, specialists…
MLD adminOctober 25, 2024
Orchard Therapeutics celebrates progress in advancing newborn screening for MLD on International Neonatal Screening Day
Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of…
MLD adminJuly 15, 2024
Clinical trial update from Takeda
Since the announcement that the phase 2 trial with TAK-611 for children with Metachromatic Leukodystrophy…
MLD adminMarch 20, 2024
A Rare Find: The Newborn Screening Collaborative comedy short
The UK currently screens newborns for just nine rare but serious conditions whereas the USA…
MLD adminDecember 4, 2023
A Rare Find: Comedy short wants to create conversation around newborn screening
The UK currently screens newborns for just 9 rare but serious conditions whereas the USA…
MLD adminNovember 22, 2023
Genomics England includes MLD on its provisional list
The Generation Study, a project by Genomics England, will see the genomes of 100,000 newborns…
MLD adminOctober 4, 2023
Gene Therapy Study for Late Juvenile Metachromatic Leukodystrophy (LJ MLD)
Following the wonderful news that Gene Therapy was approved for use in England by the…
MLD adminFebruary 22, 2022
NICE final guidance approves life-changing gene therapy for treating Metachromatic Leukodystrophy (MLD)
We are delighted to announce that Atidarsagene autotemcel (Libmeldy®) has been recommended as an option…
MLD adminFebruary 4, 2022
Newborn Screening for MLD Update
A formal application to add Metachromatic Leukodystrophy to the UK Newborn Bloodspot (NBS) programme has…
MLD adminDecember 9, 2021
Update on approval of gene therapy for MLD
NICE (National Institute of Clinical Excellence) the drug decision making body have publicly released their…
MLD adminJuly 14, 2021