A Wiltshire family whose two-year-old daughter has been diagnosed with a very rare and life-limiting genetic condition say they hope to make the most of the time they have left with her.
Natasha Bowen-Ashwin and her husband Richard launched an online fundraising appeal which has so far raised more than £5,000 – to buy equipment to make life easier for their youngest child Penelope.
The toddler was diagnosed in May with Metachromatic Leukodystrophy (MLD), a very rare, life-altering and life-limiting genetic condition, following an enzyme blood test after her parents noticed her physical development had begun to regress.
The heartbroken couple, who live in Snarlton Close, Melksham, and have an elder daughter Tabitha, aged five, were devastated to learn that Penelope has the most aggressive form of the disease 'In late-infantile MLD’.
Natasha, 31, said: “Penelope is a gorgeous, smiley, happy little girl. She loves playing with her babies, looking through books, especially ones with flaps. Her absolute favourite is music and doing the actions to her favourite songs.
“Penelope loves being out and about and taking in the world around her. She really enjoys being around animals and having cuddles with them.
“She is super affectionate and loves her family and although she struggles with her words has learnt to sign 'love you'."
She said the family are heartbroken but want to make the time left withy her as "memorable as possible and create special memories with her sister Tabitha".
She added: “It is very likely we will require additional equipment to support her along the way to keep her happy and comfortable. We want to take her places and do things, so she can experience as much of life and the world as possible.”
After a period of normal growth and development, Natasha said Penelope started to experience difficulty when crawling or walking and trying to lift herself up.
This usually happens at 15 to 24 months. Further deterioration then occurs and most children with this form of MLD generally die by age five.
Natasha said: “The ability to take food by mouth will be lost, due to loss of control swallowing and she will require a feeding tube into her stomach.
“We have been told that the most rapid deterioration will happen over the next year between the ages of 2 to 3 years.
“Crushingly, Penelope isn't eligible for the life-saving gene therapy treatment as she already has nerve damage,” Mrs Bowen-Ashwin said.
“The specialists can't say how long we have left with Penelope, just that we should make the most of our time with her and do as much as we can whilst she is still mobile and strong enough.
“Many medical professionals, parents of children lost and parents currently going through this awful disease are lobbying for heel prick tests at birth.
“If this condition is caught early, before any damage is done then more children can have life-saving gene therapy.”
To donate to the appeal, visit gofundme.com/f/penelopes-mld-journey?.
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