Calling all carers of Late-Infantile MLD sufferers

A major pharmaceutical company are trying to move their Gene Therapy Research & Development program forward by gaining an understanding of the impact of the rare disease in question (Metachromatic Leukodystrophy-MLD) in patients and their carers, as well as the overall treatment pathway.

To further develop the gene therapy medicines they are looking specifically for the emotional, physical, societal and economic impacts, as well as the disease journey pre- and post-diagnosis. They would also like to know which changes in disease management would make the most meaningful differences.

The only criteria would be that the first symptoms appeared between birth and 30 months, i.e. late infantile onset MLD. They can have received a stem cell transplant or taken part in a clinical trial but not GSK’s gene therapy or scheduled to receive gene therapy within the next 12 months.

To get this information they are carrying out series of interviews with carers (and patients when suitable), either in their homes or over skype. They are happy to share a summary of the findings with our charity as a thank you for our help.

Please contact us if you are willing to take part in this research. Email

Category: Blog · Tags:

Leave A Comment

About us

MLD Support Association UK was set up to bring hope to families in the fight to eradicate Metachromatic Leukodystrophy (MLD). Our aim is to provide support by way of shared information from people in similar circumstances who have already experienced the effects of the condition and/or any treatments available.

MLD Support Association UK

MLD Support Association UK is a
Registered Charity Number 1150542

Professor Timothy M Cox, MD, FRCP, FMedSci
Suzi Digby (Lady Eatwell) OBE
Iain Stewart MP


MLD Support Association UK
Floor 5
Amphenol Business Complex
Thanet Way

Tel: 07977 440809