<div>Our beautiful little Leni, who is just three years old and so full of love and life, has been diagnosed with an extremely rare and very severe rapidly neurodegenerative genetic condition called <strong>Sanfilippo Syndrome Type B (MPSIIIB)</strong>, also known as<strong> Childhood Dementia</strong>. Life-saving treatment may be available this year, but we need to urgently raise funds and awareness for her to access this. With Leni’s condition every day matters and <strong>time</strong> <strong>is</strong> <strong>running</strong> <strong>out</strong> <strong>to</strong> <strong>save</strong> <strong>her</strong> <strong>life</strong>.
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<div>Children with this condition have a defect in a single gene that prevents them from breaking down a sugar molecule called heparan sulfate, causing toxic waste to build up in the brain and body.
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<div><strong>Sanfilippo</strong> <strong>Syndrome</strong> <strong>is</strong> <strong>unimaginably</strong> <strong>cruel</strong>. Children develop relatively ‘normally’ with no obvious symptoms until they are around two years old, then over a short amount of time they lose all skills once learned, experience severe behavioural challenges, extreme hyperactivity, insomnia that can last for days, seizures, a total loss of cognitive and motor function, and a premature death in their early to mid teens, as well as many other horrific symptoms. <strong>Sanfilippo is relentless and leaves nothing untouched</strong>.
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<div><strong>Leni will lose the ability to walk, talk, play, laugh, eat, and eventually her life to this condition.</strong>
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<div>Some of our wonderful friends are completing gruelling challenges to help raise the funds and awareness needed to prevent this from happening. Every donation and share is another step closer to life-saving treatment for Leni:
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<li><strong><a href="https://www.gofundme.com/f/14-miles-for-leni-every-step-counts" target="_blank" rel="noreferrer noopener">HalfMarathon4Leni</a>- </strong>A half marathon along the Cornish coast - from Harlyn Bay to Newquay - by Leni's Godmother Charlotte
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<li><strong><a href="https://www.gofundme.com/f/running-50-miles-to-save-leni" target="_blank" rel="noreferrer noopener">50Miles4Leni</a>- </strong>The Testway 50 Ultra Marathon by Gemma
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<div>Like most of the world, we had never heard of Sanfilippo Syndrome until Leni's diagnosis and it is now part of every waking moment and has <strong>ripped our</strong> <strong>world apart</strong>. No parent should have to go through this heartbreak, and no child should have to suffer with this syndrome, especially when <strong>the science already exists to prevent symptoms</strong> when the child is treated early enough.
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<div><strong>There is currently</strong> <strong>no approved treatment and no cure</strong>, but since launching Leni’s campaign there are now multiple promising <strong>experimental treatments on the very near horizon</strong> that have already been proven to be incredibly effective. Everyone is working very hard to make these treatments urgently accessible.
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<div><strong>We will do whatever we can to get Leni and other children access to the treatment that they desperately need, but at the moment it is <em>just</em> out of reach - partly due to lack of funding.</strong>
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<div>With Leni’s condition days, weeks and months matter as <strong>toxic waste builds up in her tiny body every single day</strong> and the damage cannot be reversed once it is done. Time is working against us. The difference between her getting access to this treatment now and in a years time could be the difference between a potentially near-normal life and a significantly shortened life with the most severe mental and physical disabilities you can imagine. <strong>It’s</strong> <strong>now</strong> <strong>or</strong> <strong>never</strong> <strong>for</strong> <strong>Leni</strong>.
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<div><strong>It</strong> <strong>is every parent’s worst nightmare to be told that their child is going to die after years of suffering</strong>, and that is the haunting reality we are faced with if Leni cannot get access to treatment and fast. We will do anything we can to save our baby girl, and that starts with raising awareness and funds to get her access to life-saving treatment. <strong>This</strong> <strong>truly</strong> <strong>is</strong> <strong>her</strong> <strong>only</strong> <strong>hope</strong>.
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<div><strong>We know that the best chance of us getting Leni and other children access to this treatment is by campaigning and fundraising</strong>, but the exact pathway is unclear.
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<div>There are multiple potential clinical trials expected to start within the next 12 months and we are engaged with all involved parties from around the world. We will keep you updated as we know more.
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<div>If we are unsuccessful in accessing these specific experimental treatments then funds will go towards any future treatments that may become available, to providing for her complex medical needs, and to improving her quality of life wherever we can. We will also donate funds to <strong>Great Ormond Street Hospital </strong>who are providing Leni with truly world-class care, and the <strong>Cure Sanfilippo Foundation</strong> who have been a huge support for us and many other children and families.
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<div>We are also working closely with the <strong>MPS Society</strong> &amp; the <strong>LSD Collaborative</strong> on '<strong>Childhood Dementia UK</strong>' - a new initiative created to raise the public visibility of Childhood Dementia, including Sanfilippo Syndrome, and to fight for its inclusion in government policy &amp; planning. Some of the money raised will be used to help support this initiative, with the hopes of securing sustainable access to treatment for Leni and other children, and for Sanfilippo to be included in the newborn screening so no future family has to go through what we have been through.
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<div>We have published a children's book called 'Saving Leni' - with every penny raised from the sales of this book going directly to Childhood Dementia UK. <strong><a href="https://amzn.eu/d/0gzhLheW" target="_blank" rel="noreferrer noopener">'Saving Leni' is available to purchase here</a>. </strong>
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<div>We will be sharing updates on Leni’s journey on her Instagram <strong><a href="https://www.instagram.com/saving_leni" target="_blank" rel="noreferrer noopener">@saving_leni</a></strong>, TikTok <strong><a href="https://www.tiktok.com/@saving.leni" target="_blank" rel="noreferrer noopener">@saving.leni</a></strong>, and here on her GoFundMe page.
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<div>We have been completely overwhelmed by the love and support we have received since Leni’s diagnosis, we are so so grateful and we know that <strong>she is so loved</strong>.
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<div><strong>Leni's story has received extensive coverage in the national press. We've shared some highlights below where you can learn more about her condition and her fight to access life-saving treatment:</strong>
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<li><a href="https://www.thetimes.com/life-style/parenting/article/childhood-dementia-experience-vhr880drt" target="_blank" rel="noreferrer noopener">The Times</a>
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<li><a href="https://www.independent.co.uk/news/health/sanfilippo-childhood-dementia-leni-forrester-b2946332.html" target="_blank" rel="noreferrer noopener">Independent</a>
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<li><a href="https://www.mirror.co.uk/news/uk-news/our-little-girl-2-dementia-36903755" target="_blank" rel="noreferrer noopener">The Mirror</a>
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<li><a href="https://www.dailymail.com/health/article-15702695/Our-daughter-childhood-dementia-no-obvious-signs.html" target="_blank" rel="noreferrer noopener">Daily Mail</a>
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<li><a href="https://people.com/2-year-old-girl-diagnosed-rare-childhood-dementia-11932006" target="_blank" rel="noreferrer noopener">People Magazine</a>
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<div>We know that it is a big ask, but please donate if you are able, and please share Leni’s story and GoFundMe page everywhere you can to help raise the awareness and funds needed to <strong>save Leni’s life</strong>.
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