Research 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
Research Newborn Screening for MLD Update A formal application to add Metachromatic Leukodystrophy to the UK Newborn Bloodspot (NBS) programme has…MLD adminDecember 9, 2021
Research 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
FamiliesResearch MLD UK registry news Following long discussions around the current worldwide status of Registries for MLD and because of…MLD adminJuly 12, 2021
FamiliesResearch Treating early onset MLD We are pleased to report Orchard Therapeutics has received EC full (standard) market approval for…MLD adminDecember 21, 2020
FamiliesResearch The Survey to understand the MLD patient and care-giver experience is now live This research is extremely important for us to gather, so we can ensure that regulatory…MLD adminAugust 28, 2020
AllFundraisingResearch Making a leap for rare diseases Metachromatic Leukodystrophy (MLD) is classified as a Rare Disease There are over 300 million people…MLD adminFebruary 27, 2020
AllResearch A Study of Intrathecal SHP611 in Participants with Late Infantile MLD A new Clinical Trial has been announced by Shire (now part of Takeda Pharmaceutical Company…MLD adminDecember 14, 2018
AllFamiliesResearch Family Conference The second MLD Support Association UK Family Conference was held at Wyboston Lakes in St.…MLD adminJune 12, 2018
AllResearch Scientific Conference On Friday 8 June 2018, we held our second Scientific Conference at Wyboston Lakes in…MLD adminJune 11, 2018