Saving Leni

▶

▶ 10.8K ❤ 0 💬 0

I find myself wishing, more than anything, that I could freeze time. At the moment Leni is just like any other two year old, and I wish I could keep her exactly as she is right now. Keep her little laugh, her words, her personality, her love of slides, and all the little things that make her beautifully her. I wish I could protect her from everything that is coming. Before differences between her and her friends become more obvious, before she starts to lose the skills she has worked so hard to learn - not overnight, but slowly, one by one. Things that once came so naturally will become difficult. Words will get harder to find. Little routines will become unfamiliar. Eventually she won’t recognise us. Leni deserves to grow, not lose. To keep learning, discovering, and becoming the incredible little person she is. We can’t freeze time. So instead we are fighting. Fighting for a different future for Leni and for all children with dementia where they keep growing and get the chance to live. #dementia #motherdaughterlove #toddlertok #specialneedsparenting #cherisheverymoment

▶

▶ 120.7K ❤ 0 💬 0

This week the Sanfilippo community has received the INCREDIBLE news that the first ever treatment for Sanfilippo Type A has been approved ✨ The FAYUVI Gene Therapy (also known as UX111) from Ultragenyx has the potential to slow the devastating, irreversible neurological progression and loss of function associated with this heartbreaking condition, and in the youngest children it has the potential to stop it completely. It’s a miracle, and one that clinicians, researchers, patient associations, charities, pharmaceutical companies and families have been fighting for for over a decade. We couldn’t be happier for our Type A friends, and whilst we feel a deep sadness that this approval has come far too late for many children despite this treatment existing for many years, it gives us hope for the future 🤍 So… what does this mean for Leni? Many of you have reached out sharing this news, and we appreciate it so much 🫶🏻 Unfortunately Leni will not be able to have this treatment, for a couple of reasons… 1. All treatments are type specific - this treatment is for children with Sanfilippo Type A, and Leni has Type B. 2. This treatment has only been approved by the FDA - which means at the moment it is only available in the US. International patients will need to self-fund with a price tag of $3.95 million. Although Leni isn’t able to have this treatment, we hope it is a sign of positive things to come for the wider Sanfilippo community and for Leni ✨ #sanfilippo #raredisease #sendparents #toddlertok #toddlersoftiktok

▶

▶ 9.1K ❤ 0 💬 0

Individually rare, collectively as common as Childhood Cancer - but no one knows it exists. We wanted to shine a light on some of the brave, beautiful children with dementia, and the heartbreaking conditions they are battling. Over 50,000 babies are born with one of the 145 different genetic conditions that cause Childhood Dementia every year. All of these conditions are terminal, taking a life every 11 minutes, with an average life expectancy of just 10 years old. Childhood Dementia is progressive. Children are often born apparently healthy then over time lose the ability to walk, talk, run, play, eat, drink & eventually their lives. It’s cruel, relentless & devastating. There are no approved treatments for many Childhood Dementia-causing conditions anywhere in the world & over half have no active clinical trials. We are fighting to change that, and together we can save these brave, beautiful children… 🤍 Sadie, Sanfilippo Type A, @Sadie & Fam 🤍 Henry, Hunter 🤍 Remi, Rett 🤍 Lottie, Sanfilippo Type A & Down Syndrome 🤍 Liam, Niemann Pick Type C 🤍 Grant & Hazel, MCAHS1, @Hazel Mae-Medical 🤍 Liv, Sanfilippo Type B, @Saving Liv 🤍 Charli, Batten 🤍 Poppy & Oliver, Sanfilippo Type B, @Cure Poppy + Oliver 💜 🤍 Benjamin, Hunter 🤍 Veda, Sanfilippo Type A, @Saving Veda 🤍 Cody, Sanfilippo Type A, @codys_fight 🤍 Nellie, MLD 🤍 Clark, Sanfilippo Type A, @Cloey | Sanfilippo Mama 💜 🤍 Sophia, Sanfilippo Type A 🤍 Bertie, BPAN, @Bertie’sBpanJourney💙 🤍 Scarlett, BPAN, @Scarletts BPAN Battle 💗 🤍 Emilia, Sanfilippo Type A 🤍 Elle, Sanfilippo Type C, @Team Elle 🤍 Nikolai, Hunter 🤍 Julek, INAD 🤍 Merrick, Sanfilippo Type A, @Hope for Merrick 🤍 Noah, Niemann Pick Type C Please, please share 🙏🏻 #dementia #dementiaawareness #raredisease #specialneedskids #specialneedsparenting