Fundraiser for Tooting girl with Childhood Dementia
At age 2, Tooting-born Leni Forrester has been diagnosed with Sanfilippo Syndrome Type B (MPSIIIB), also known as Childhood Dementia, an extremely rare & very severe neurodegenerative genetic condition.
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 & body.
There is currently no treatment or cure, but there is a promising experimental Enzyme Replacement Treatment that has been proven to be incredibly effective. The company that owns the licence to this treatment is working hard to make this treatment urgently accessible to those who need it.
Raising £200,000 is the only way for the family to potentially access the treatment – you can donate to help the Forrester family reach their target via the GoFundMe page.
Children with Sanfilippo Syndrome Type B lose (in a short pace of time) all skills once learned, experience severe behavioural challenges, hyperactivity, insomnia, seizures, a total loss of cognitive & motor function, & a premature death in their early to mid teens, as well as many other symptoms.
Leni’s mother Emily said: “Leni will soon lose the ability to walk, talk, laugh, eat, & then eventually her life to this condition.”
“With Leni’s condition weeks & months matter as the damage cannot be reversed once it is done. The difference between her getting access to this treatment now & in a year could be the difference between a near-normal life & a significantly shortened one with the severe mental & physical disabilities.”
If unsuccessful in accessing this specific treatment, then funds will go towards any future treatments that may become available, providing for Leni’s complex medical needs, & improving her quality of life. The family will also donate funds to Great Ormond Street Hospital and Charity which is providing Leni with world-class care, & Cure Sanfilippo Foundation, the only charity dedicated to Sanfilippo Syndrome.
Updates on Leni’s journey can be seen on @saving_leni (Instagram) & on her GoFundMe page.