Research

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

Families Research

MLD UK registry news

Following long discussions around the current worldwide status of Registries for MLD and because of…

MLD adminJuly 12, 2021

Libmeldy EU approval for treatment of early onset MLD

Families Research

Treating early onset MLD

We are pleased to report Orchard Therapeutics has received EC full (standard) market approval for…

MLD adminDecember 21, 2020

Families Research

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

Rare Disease Day 2020. MLD Support Association UK

All Fundraising Research

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

All Research

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

All Families Research

Family Conference

The second MLD Support Association UK Family Conference was held at Wyboston Lakes in St.…

MLD adminJune 12, 2018

All Research

Scientific Conference

On Friday 8 June 2018, we held our second Scientific Conference at Wyboston Lakes in…

MLD adminJune 11, 2018

NICE final guidance approves life-changing gene therapy for treating Metachromatic Leukodystrophy (MLD)

Newborn Screening for MLD Update

Update on approval of gene therapy for MLD

MLD UK registry news

Treating early onset MLD

The Survey to understand the MLD patient and care-giver experience is now live

Making a leap for rare diseases

A Study of Intrathecal SHP611 in Participants with Late Infantile MLD

Family Conference

Scientific Conference

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