Following the wonderful news that Gene Therapy was approved for use in England by the NHS for pre-symptomatic Late-Infantile and pre-symptomatic and mildly-symptomatic Early Juvenile MLD, we now have more exciting news! A clinical study for sufferers of pre-symptomatic and early-symptomatic sufferers of Late Juvenile MLD is now recruiting. The study is being conducted at Ospedale San Raffaele – Telethon Institute for Gene Therapy (OSR-TIGET) in Milan, Italy.NEWS PAGE
We are delighted to announce that Atidarsagene autotemcel (Libmeldy®) has been recommended as an option by NICE (The National Institute for Health and Care Excellence) for treating metachromatic leukodystrophy (MLD) in England and Wales for eligible children. Children eligible are:
- children who have late infantile or early juvenile types, with no clinical signs or symptoms
- children who have the early juvenile type, with early clinical signs or symptoms, and who can still walk independently and have no cognitive decline.
NICE committee members concluded that “MLD is a rare, serious, and life-limiting condition that significantly affects the lives of people with the condition, their families and carers.” They commended the patient organisations for their submissions providing detailed feedback from a survey on the effect of Libmeldy® on quality of life. They also acknowledged that Libmeldy®, manufactured by Orchard Therapeutics, is an innovative technology and represents a step-change in managing MLD.
Vivienne Clark, Chair of The MLD Support Association UK, said: “We are delighted with today’s announcement from NICE confirming that Libmeldy® has been recommended for use in the treatment of MLD for pre-symptomatic Late infantile or Early Juvenile and early-symptomatic Early Juvenile children. This is a major development in our fight to eradicate MLD and we would like to wholeheartedly thank all the stakeholders, clinicians and researchers for their continued hard work and dedication in making this treatment a reality. We would also like to acknowledge all the affected children and their families whose lives have been affected by this condition – many of whom selflessly supported our research despite no possibility of benefitting from this treatment – their courage, bravery and strength has played the greatest role in this achievement.”
Georgina Morton, CEO of ArchAngel MLD Trust and patient expert, said: “We would like to thank NHS England and NICE for recognising the immeasurable suffering of MLD patients and the life-changing potential of Libmeldy®. This recommendation is a salute to the tireless commitment of many clinicians, advocates, investors and affected families and heralds a pivotal move towards vanquishing this appalling disease. It also facilitates an important step closer to newborn screening for MLD and a future which allows all eligible patients to access this remarkable treatment at the earliest opportunity.”
Nicola Elson, a patient expert, said: “We are completely overjoyed with the decision from NICE to recommend Libmeldy® for use in England and Wales for eligible children. With an MLD diagnosis for two of our children, one untreated and the other seven years post-transplant, we have witnessed first-hand the pain, torture and devastation this monstrous condition unleashes on innocent children. We have also seen the results of this ground-breaking treatment and the second chance at life it brings. We are thrilled those other children will have access to this therapy.”
Orchard Therapeutics CEO, Professor Bobby Gaspar, M.D., PhD said: “Today’s landmark decision by NHS England follows a thoughtful and comprehensive value assessment by NICE and represents a major milestone for the MLD community, Orchard, and the entire field of HSC gene therapy”.
Bob Stevens, Group Chief Executive of The MPS Society, said: “The decision by NICE to recommend Libmeldy® marks a milestone in the evolution of treatments and therapies for rare metabolic conditions. It is the first time that gene therapy has been recommended in England for any of these diseases and what was once science fiction has now become science fact. Today as a result of collaboration, we have another treatment option to add to the existing innovative therapies for one of our diseases, MLD. Today, hope grew a little larger for our community.”
The final evaluation document (FED) can be found here.
When NICE recommends a treatment the NHS must make sure it is available within 3 months.
We would like to thank all the MLD community, clinicians and patient groups for all their submissions, representation at committee meetings and information shared through the surveys and personal stories.
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Orchard Therapeutic’s press release can be found here
About the Patient organisations:
Thanks to National Lottery funding the MLD Support Association UK Funday will take place on Sunday 10th April at Drayton Manor (Tamworth, Staffordshire, B78 3TW).
Drayton Manor Resort consists of a theme park with over 100 rides and attractions including plenty of family fun rides, Europe’s only Thomas Land, and a 15-acre zoo. The event is open to all UK MLD families and will include tickets into the park, refreshments and a light lunch.
To register for the Funday please complete the form below by Friday 18 February. If you have any questions please do get in touch.
Drayton Manor’s objective is to ensure the site is accessible to all guests. Further information and guidance can be obtained from guest services, or from our web site, or in literature upon request. Information is also indicated on primary safety instruction signs at each ride/attraction.
Nursing mums and baby changing
Nursing Mothers and Baby Changing facilities are located opposite the Safari Pizza and Pasta. Baby changing facilities can be found in all of our toilet facilities.
Manned by qualified staff which is located in the centre of the park. Situated next to the 4D Cinema.
Accessible toilets and changing rooms
Drayton Manor Park have installed a brand-new Hoist Room. This brand-new room consists of a toilet, a wash hand basin, a shower, a hydraulic bed and a hoist. Most importantly the room is spacious enough to provide ample room for carers and is located behind the First Aid department. The electronic hoist is easy to use with a foot pedal to pump it up to its maximum level above waist height and the environment surrounding this area has been made easily accessible and wheelchair friendly. All the relevant support bars, as well as an emergency call rope, have been installed to ensure users can be supported at all times. Please note: Customers are asked to bring your own sling.
A formal application to add Metachromatic Leukodystrophy to the UK Newborn Bloodspot (NBS) programme has now been made to the National Screening Committee (NSC). This is an important milestone for the MLD community and one which could not have been possible without the important support and ongoing collaboration of many brilliant scientists and heroic clinicians.
Unfortunately this will be a lengthy and challenging process, as the UK NSC is in urgent need of reform. We will continue to work hard for future MLD affected families and to campaign for changes to the system which will bring hope to many other rare diseases where treatments and tests are available.NEWS PAGE
Great News! We will be holding a virtual Christmas Party on Saturday 11th December 1pm-2pm for our MLD children and their siblings. We will also be distributing Festive Family packages in the lead up to Christmas to UK MLD families. If you would like to register your family, please follow the link to complete the required contact information before 7th November.NEWS PAGE
AmazonSmile is a really simple way for you to support MLD Support Association UK every time you shop, at no cost to you.
The way it works is that Amazon donates 0.5% of the price of eligible AmazonSmile purchases to charities, and MLD Support Association UK is now registered with AmazonSmile. There is no catch – it is the same Amazon you know – same products, same price and same service.
So please register with AmazonSmile and help MLD Support Association to continue providing support to families affected by MLD. There are millions of eligible products available – look out for ‘Eligible for smile.amazon.co.uk’ on the product’s details page.GET STARTED
Great News! We have secured funding from the National Lottery Community Fund to hold a 2021 Christmas Party – Date and time to be confirmed in the near future. The last eighteen months have been tough on everybody, but especially our little ones, siblings, parents and carers and it will be wonderful to get together at this special time of year.
As an organisation we will be monitoring the Covid-19 situation closely, working within the government guidelines and potentially adding a few of our own in order to keep all our VIPs as safe as possible. #NationalLottery,NEWS PAGE
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 Metachromatic Leukodystrophy (MLD).
Although NICE have acknowledged the clinical benefit to patients they have given a “NO” decision due to long term uncertainty. This is not NICE’s final decision and the committee is proposing to meet again on the 6 October 2021 following review of the feedback from consultees and members of the public.
Members of the public have until the 30th July 2021 to submit views and feedback to NICE. The evaluation committee will then reconsider the evidence and their decision at the proposed meeting in October.Download Press Release
Following long 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.
It is extremely important to have a National Database of all known families in order to keep them updated on developments, and to be able to contact them. We have, therefore, decided to create the National MLD Families Database which will be held by MLD Support Association UK, strictly adhering to GCP guidelines
It will be safe and secure.
When we have more details we will let you all know and post information on how to consent to participate.BACK TO NEWS PAGE
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. At the moment 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.
BIMDG is the professional society for health professionals involved in the diagnosis and management of individuals with inherited metabolic disease, which includes MLD. They have published an information sheet on COVID-19 vaccinations which can be downloaded hereNEWS PAGE
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 releaseNEWS PAGE
Exciting news! Funding from the Coronavirus Community Support Fund, distributed by The National Lottery Community Fund, means that MLD Support Association UK are able to offer Covid Care Packages to our UK superheroes. Nominations have to made by a parent or close family members and have to be in by Friday 18th September. The two care packages to choose from will be either aromatherapy or visual and sound sensory. There is just a very short form below to complete.
For further information please contact email@example.com
Care package application
This research is extremely important for us to gather, so we can ensure that regulatory bodies such as NICE and the NHS fully understand the effects of MLD on patients and families ,and that we do our best to secure access to therapies and support future access to Newborn Screening. The survey will be available until 14 September 2020.
The survey should take you no longer than one hour to complete and you are able to stop and return to it later (your answers will be saved for one week). It may be advisable to have any documents to hand before starting that will help you to answer any diagnosis or medical questions to hand.
Rare Disease Research Partners (RDRP) are conducting this survey on behalf of the MPS Society UK, MLD Support Association UK and ArchAngel MLD Trust. If you have any questions, require a paper copy or wish to do the survey over the telephone, please email firstname.lastname@example.org
Thank you in advance for your support.Access survey
Friday 10 July at 11.30am
It has been a very tough few months for all families dealing with MLD and shielding. Would you like to join us on a Family Zoom Coffee Break Meeting on Friday 10 July at 11.30am? We can have a coffee, chat and catch up. If you would like to join us please contact email@example.com for further details.
With the risk of catching coronavirus steadily decreasing, the UK Government announced changes to the future of the shielding programme. From 6 July those shielding will be able to meet outdoors, in groups of up to six people from outside their household, with social distancing:
If you live alone (or are a lone adult with dependent children under 18), you will be able to form a support bubble with another household. From 1 August, many people will no longer need to shield, and the advice will be that you can visit shops and places of worship, but you should continue to maintain social distancing whenever possible. You can find the full guidance on the GOV.UK website.
You should shortly receive a letter from your GP or Specialist Centre advising you of these changes. Please do not hesitate to contact Jackie Imrie if you have any questions about your own level of risk as people are encouraged to spend more time outdoors, and to gradually return to normal activities, school and the workplace.
We understand how challenging it has been for many of our families who have had to shield over the past few months, and we also appreciate how anxious they may be about the changes to the guidance – as well as what this may mean for them. Please keep in mind that we are here to support you should you need any further help explaining the latest shielding guidance over the coming weeks.
Metachromatic Leukodystrophy (MLD) is classified as a Rare Disease
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.
Each rare disease may only affect a handful of people, but taken together the number of people directly affected is equivalent to the population of the world’s third largest country.
Rare diseases currently affect 3.5% – 5.9% of the worldwide population.
72% of rare diseases are genetic and 70% of those genetic rare diseases start in childhood.
Lack of knowledge about rare diseases by health professionals leads to a delay in diagnosis and, frequently, it is then too late for treatment.
Rare Disease Day raises awareness for the 300 million people living with a rare disease. The Rare Disease Campaign aims to achieve equitable access to diagnosis, treatment, Health and social are and social opportunity for people affected by a rare disease.
Find out more about Rare Disease Day
Saturday 7 September dawned mild and partly sunny. It was a perfect day for the first MLD Support Association UK Family Fun Day at Wicksteed Park in Kettering. The day was a great success with rides, the little train, a magician/balloon maker, an amazing face painter and plenty of time for all the families to mingle. A great big THANK YOU to the National Lottery Community Fund for giving us a grant to help pay for the day.
“It was lovely meeting up with other families and siblings.”
Hannah Johnson, parent
“I enjoyed meeting the other families while the children had the chance to have some fun.”
Nicola Elson, parent
“The grounds were open and the venue was big enough for wheelchairs. Having a changing facility was very helpful. Being able to get a cup of tea/coffee whenever I wanted and having food available for a long time was good as it’s tricky to eat and drink within certain time frames. Also interaction between siblings and parents was great! The staff were very helpful and the venue was central so many people could attend.”
Paula Veysey, parent
“I loved meeting all of the families and getting to know the day-to-day things about each of us.”
Tina Maii, parent
“The event was really well organised. The kids loved the entertainment and so much thought and effort went into their gifts. Loved the beach theme and it was lovely meeting up with all the other families.”
Twila D’Cruz-Parsley, parent
Keiren Martin completed his Abseiling challenge from the ArclorMittal Orbit in August 2019. He regularly takes on challenges for charity, and this time has chosen. It is amazing that he has raised almost £500.
We are delighted to announce we have been awarded a National Lottery Community grant of over £4,000 to fund our Family Fun Day at Wicksteed Park in Northamptonshire on 7 September 2019. A big thank you to our Trustee Nicola for securing the grant, which provides us with the means to invite affected children and adults, siblings, mums and dads and grandparents who help care and also carers, where appropriate.
As well as enjoying a great day out, our aim is to bring together people from all walks of life who are affected by MLD to share their experiences and continue building our support network. We will be providing free entry (wristbands) to the Park, and we have a private room for a light lunch for everyone.
If you would like to register your interest in our Family Fun Day, please email firstname.lastname@example.org
The National Lottery Community Fund continues to support good causes which improve lives by bringing people together and building strong relationships in and across communities. You can find out more about their work here.
Andrew Hudson recently learnt about a young man suffering from MLD and realised just how few people know about this rare disease and wanted to support our charity to raise awareness, support families and fund research. He will be running his 10th Brighton Marathon in April and thought it would be a good idea to up his game and try and raise a decent amount of money. He wrote on his Just Giving page, “People like me take good health for granted but many people have their health stolen by cruel life changing illnesses like MLD”. Thank you Andrew for choosing to run for us.
Coded to Pixel Perfection
Are you ready to show off & make some jaws drop?