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in the fight to eradicate MLD

 
 
 
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Gene Therapy Study for Late Juvenile Metachromatic Leukodystrophy (LJ MLD)

By Uncategorized
Gene Therapy Study for Late Juvenile MLD

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.

Download full details on the gene therapy study.

NEWS PAGE

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

By Research

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.

– END –

Other information:

Orchard Therapeutic’s press release can be found here

About the Patient organisations:

MLD Support Association UK
www.mldsupport.org.uk
Contact
Vivienne Clark (Chair)
vivienne@mldsupportuk.org.uk

ArchAngel MLD Trust
www.archangel.org.uk
Contact
Georgina Morton (CEO)
contact@ArchAngel.org.uk
georginamorton@archangel.org.uk

The MPS Society
www.mpssociety.org.uk
Contact
Sophie Thomas (Senior Head of Patient Services and Clinical Liaisons)
s.thomas@mpssociety.org.uk

Announcing Family Fun Day at Drayton Manor Resort

By Families, Support
 
 
 
 
 
 
 
 

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.

Disabled access

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.

Medical

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.

Registration form

Newborn Screening for MLD Update

By Research

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. 

Please visit ArchAngel.org.uk/NBS or email contact@archangel.org.uk for further details.

NEWS PAGE
MLD Support Association UK Christmas Party

Christmas party announcement

By Families, Support
MLD Support Association UK Captain Fantastic Christmas Party

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

Support us with Amazon Smile

By Fundraising
Amazon Smile MLD Support Association UK

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.

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mld-support-association-uk-christmas-party

Christmas party 2021

By Families, Fundraising, Support

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
MLD Gene Therapy update NICE

Update on approval of gene therapy for MLD

By Research

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
MLD Patient Registry

MLD UK registry news

By Families, Research

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.

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MLD Support Association UK COVID-19 Vaccinations

COVID-19 vaccinations for people with MLD

By Uncategorized

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 here

NEWS PAGE
MLD Support Association UK Car Packages

Lottery funding secured for care packages

By Families, Support
Care packages for MLD sufferers

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.

Thanks to the Government and The National Lottery Community Fund. @TNLCommunityFund  @dcmsgovuk  @TNLComFund and @DCMS #CommunitiesCan.

For further information please contact vivienne@mldsupportuk.org.uk

Care package application

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

By Families, Research

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 info@rd-rp.com

Thank you in advance for your support.

Access survey
MLD Support Association UK COVID-19

COVID-19 update on shielding guidance

By Families, Support

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.

Rare Disease Day 2020. MLD Support Association UK

Making a leap for rare diseases

By All, Fundraising, Research
MLD Support Association UK proudly supporting Rare Disease Day

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

MLD Support Association UK Family Fun Day 2019

Family Fun Day at Wicksteed Park

By All, Families, Support
 
 
 
 
 
 
 

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

Celebrating National Lottery Community Funding

By All, Fundraising
 
 
 
 
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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 admin@mldsupportuk.org.uk 

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 Runner

Andrew Hudson learns of MLD sufferer and decides to support our work

By All, Fundraising

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.

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