Help Fund Mylah’s Treatment To Walk Again!

Urgent Appeal for Mylah: Help Mylah Beat Arginase Deficiency and help get her mobility back!!

Our sweet daughter Mylah, a brave three-year-old, is facing an incredibly tough battle. She was diagnosed at birth with a rare and devastating condition called Arginase Deficiency, part of the Urea Cycle. This severe metabolic disorder is relentlessly attacking her health, causing her mobility to deteriorate day by day. She has followed a strict low protein diet and is under the fantastic Alder hey/ Manchester Children's Metabolic team.

Although Mylah has this awful condition, she had been developing well. she was meeting all milestones and was doing very well. However, around May this year, she began struggling to walk, and within just a couple of weeks, lost the ability to walk and stand altogether. The rapid progression of Arginase Deficiency has been both shocking and heart-breaking, robbing her of the simple joys of childhood mobility. What once were easy, everyday moments—dashing around the room or playing in the garden—have quickly slipped out of reach.

We have also noticed it’s getting harder for her to talk—her words are coming out slower now, and sometimes she just can’t say what she wants to. It is beyond heart-breaking seeing her try so hard to tell us things. We don’t get those easy chats and giggles like before, and it’s all because her illness keeps getting worse.

Mylah is currently on the waiting list for a liver transplant under Leeds Children’s Hospital and has been since January 2024. We thought we had enough time to get the transplant before any symptoms stared, especially as she was doing so well. My uncle has amazingly put himself forward to be a liver donor for Mylah and is thankfully a blood group match, however the full assessment takes time. On top of this, due to recurring tonsilitis, her liver team want her to have her tonsils removed before the transplant. This is to eliminate the risk of tonsilitis post-transplant when she will be heavily immune suppressed.

The sad reality is that the crucial treatment, Pegzilarginase, Mylah desperately needs to slow the progression of her disease and potentially reverse it, is not currently available on the NHS. This vital enzyme replacement therapy comes at an overwhelming cost of £4,600 per vial, almost £56,00 for 12 weeks. We hope one vial per week will be enough, however we wont know for sure until she begins treatment. Without it, Mylah's condition will continue to worsen, leading to irreversible damage. The enzyme replacement therapy has been proven to work through clinical trials and has been approved in many countries around the world.

I contacted the pharmaceutical company to apply for compassionate access, but my request was unsuccessful. Mylah's fantastic metabolic doctor applied directly to Alder Hey hospital for Individualised funding last week, and today we got the devasting news that they refused to fund her treatment.

Setting up a crowd funder was the last resort, one we wished it wouldn’t have come to.

We are reaching out to you with an urgent plea for help. We are hoping to raise enough for 12 weeks initially in the hope we will have obtained funding to continue after this period if needed. Every single pound raised will go directly towards providing Mylah with treatment she so desperately needs, offering her a chance to live a more comfortable life while she waits for her transplant.

Please, if you can, contribute to Mylah's fund. No amount is too small, and every donation brings us closer to giving her the fighting chance she deserves. Please also share with as many people you possible can. Time is not on our side.

Thank you from the bottom of our hearts for your kindness, generosity, and support during this critical time. Together, we can make a difference for Mylah.

https://www.instagram.com/mylahs_journey?igsh=MTF0eHllNXJmZTIxbQ%3D%3D&utm_source=qr