MLD is a rare disease and many health professionals know little about the symptoms and outcomes. MLD Support Association UK is committed to providing information to help with a correct diagnosis and to provide details of on-going care or treatment options.
Information for healthcare professionals
We urgently need donations so that we can continue to support families and help to find a cure for MLD. If you have been affected by MLD or know a family that has and would like to make a donation, volunteer or fundraise for us, then we can point you in the right direction.
Support our work
MLD (Metachromatic Leukodystrophy) is a rare genetic condition, but when it strikes it brings devastation to the entire family. Our primary aim is to provide support to families, personally, through our Website and Facebook group and at annual Family Conferences and Fun Days. As MLD is a rare disease, many health professionals know very little about the symptoms and outcomes. We are committed to providing information to ensure a correct diagnosis and also to provide current details of on-going care or treatment options. MLD Support Association UK also organises and sponsors a bi-annual Scientific Conference where scientists and clinicians who specialise in Leukodystrophies, both from the UK and overseas, can meet up to learn about current treatment and research.
Read our family case studies
News and Stories
NICE (National Institute of Clinical Excellence) the drug decision making body have publicly released their interim decision today (9 July 2021) not to recommend OTL-200 (Libmeldy), the treatment for individuals suffering from both, late infantile and early juvenile MLD. This is not NICE’s final decision and the committee is proposing to meet again in October. Full story and press release
Following discussions around the current worldwide status of Registries for MLD and because of the unforeseen delays in getting the UK registry up and running, it has been decided to change our plans. We have decided to create the National MLD Families Database which will be held by MLD Support Association UK, strictly adhering to GCP guidelines. Read on
For people with MLD, the British Inherited Metabolic Disease Group (BIMDG) has stated that either of the COVID-19 vaccinations offered is considered safe for all inherited metabolic disorders. Vaccination is only offered to those 16 and over, and if you have any worries we suggest you contact your Consultant or metabolic unit for clarification. Download information
We are pleased to report Orchard Therapeutics has received EC full (standard) market approval for Libmeldy, an advanced therapy medicine for the treatment of early-onset MLD. A decade after the first patient was treated in clinical trials, a one-time treatment with Libmeldy has been shown to preserve motor and cognitive function. Read the full press release
There are over 300 million people living with one or more of over 6,000 identified rare diseases around the world, each supported by family, friends and a team of carers that make up the rare disease community. We are pleased to support Rare Disease Day on Saturday 29th February. Find out more
We are delighted to announce an award of over £4,000 from the National Lottery Community Fund for our Family Fun Day at Wicksteed Park on 7 September 2019. A big thank you to our Trustee Nicola for securing the grant, which will provide us with the means to invite affected children and their families. Read the full story
We have adopted a snowdrop as our emblem because it is a symbol of hope that signals the end of winter and the start of spring. Secondly, the whiteness of the flower reminds us of the brain and central nervous system’s white matter which, as a result of MLD, becomes stained and degenerates. The literal translation of MLD means (meta) change, (chromatic) colour, (leuko) white matter and (dystrophy) degeneration. About us
Millie D’Cruz Parsley celebrated her birthday in April 2010 as a normal, healthy six-year-old child. Within a couple of months, her inability to keep pace with her peers, alongside some issues with her balance, had been noticed, and her condition was eventually diagnosed as MLD (Juvenile onset). Millie’s mum, Twila, wanted to reach out and help other families with MLD, so in 2012 she donated funds to start the MLD Support Association UK. Millie’s story
Scott was born on 5 April 1972. He was healthy and happy and progressed easily through school. He was fiercely academic and at 18 he gained a place in a top UK university. Sadly, Adult-Onset MLD struck and by the age of 22 he was given a terminal diagnosis. Scott’s mother discovered that Bone Marrow Transplants were being carried out in the USA and in March 1996 Scott had a Bone Marrow Transplant with his brother, Russell, as donor. The BMT was a success and today he is the longest survivor of MLD in the UK and still remains stable. Scott’s story
We urgently need donations so that we can continue to support families, provide information for health professionals and fund research to help find a cure for MLD. Cheques should be made payable to ‘MLD Support Association UK’ and sent to the office address in Whitstable displayed at the bottom of this page.
Alternatively, you can make a donation through JustGiving. Click on the purple button and please do remember to ‘Gift Aid it’ if you are a UK tax payer. This means we can reclaim an extra 25% in tax at no extra cost to you.
Amazon donates 0.5% of the price of eligible AmazonSmile purchases to charitable organisations, and MLD Support Association UK is registered with AmazonSmile. There is no catch – it is the same Amazon you know – same products, same price and same service.
Please register with AmazonSmile and help MLD Support Association to continue providing support to families affected by MLD. Sign up to AmazonSmile