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I’m Christelle, mum to Elsie, now 18 months old. After a long IVF journey, Else was born last year with an ultra rare neurological condition, a mutation in the RARB (retinoic acid receptor) gene, there are fewer than 100 known cases in the world. There is a 1 in a 163 million chance this could have happened. You are 5 times more likely to win the lottery or get eaten by a shark .

The mutation affects the development of the nervous system and was first identified in 2013. Because of this almost all of the identified patients are children. We were completely taken by surprise as my pregnancy was healthy and every scan normal.

Elsie and I spent the first 8 months of her life in hospital and every day for her was a fight for survival. We even spent a short stint in a hospice where we went to determine whether she would live without artificial intervention - every day living on a knife edge, no one believing she would make it.

But Elsie is a fighter. We eventually came home where she has been ever since. Elsie is thriving but will have challenges. She has a visual impairment, low body tone and is currently artificially fed (she was orally fed for first 6 months of her life and we hope to get her back to this). She continues to develop all the time and has started nursery (with a carer).

We know that more advanced treatment is on the horizon and that this could change Elsie's life. It is vital to give her and other children like her the best possible outcomes as the condition is progressive.

Navigating a rare disease diagnosis is painful and bewildering. We face negativity at every turn You are told NO again and again. NO CURE, TOO RARE, NOT ENOUGH KNOWN. However, when I contacted parents of children with a similar condition, their stories were different and more hopeful. They also told me about possible treatments that are being developed globally.

ASO (Antisense Oligonucleotide) Therapy is showing the most great promise for neurological conditions like Elsie's. It treats diseases directly at the genetic level by binding to specific mRNA, either blocking the production of toxic proteins or altering how genetic instructions are read. One of the children child with the condition, is about to receive a personalised ASO therapy, a journey that began a years ago and in the USA an ASO programme to treat 200 children has been developed. Elsie would be a perfect candidate if she was American.

We want to make this possible for Elsie and other children but it needs money and we need to raise money to achieve this. Simon's family raised 1,000,000 euros to cover the cost of his treatment (creation, development, toxicity tests, trials and so on). We must create the same personalised therapy for Elsie but one that will also benefit other children, so one ASO Therapy can go further. Each time a child receives this therapy, it will bring the same thing closer to others – and make it cheaper for everyone.

Please help us achieve this and give Elsie and these other children a future to hope for. If we can make a genuine difference to their futures then Elsie will be a beacon of hope not only to me, Dan and everyone who has met her, but to the world. Elsie’s condition is rare yes but rare disease affects more than half a million people in the U.K.

Specifically, I am seeking funds to enable the amazing scientists and doctors that I have met to develop an ASO for Elsie and learn more about how they can support others. The aim is to develop a gene therapy that will improve mobility and slow down the progression of the disease, and perhaps even reverse it, as has been achieved in treatments for Huntington’s disease.

Any money not used to treat Elsie's condition will be donated more widely by the Rare People - The Research Charity for other conditions. They raise funds to support and promote high quality scientific studies that can lead to better diagnosis, improved care and crucially the discovery of effective treatments. I have joined them as a trustee.

Updates will be sent every 3 months to all who donate.