A family from Colchester, Essex has raised £86,000 to fund stem cell therapy for their one-year-old daughter who was diagnosed with myotonic dystrophy, a rare genetic condition, a month after being born weighing less than a bag of sugar. Celine’s parents, Lottie and Charlie, hope the therapy will slow the weakening of her muscles. Stem cell therapy has yet to prove effective for muscular dystrophy and has not been approved on the NHS so the family will travel to Mexico later this month for Celine’s first round of treatment, which Lottie said her daughter would need to receive every year.
Lottie said she had not expected to celebrate her daughter’s first birthday and was worried the child would be too unwell to be at home for Christmas, so she was pleased when Celine was at home on Christmas Day. She is anxious about the trip to Mexico but excited to see whether the treatment would improve her daughter’s condition. The couple’s friends and family spent the second half of last year organising fundraising challenges and events. More events were planned for 2025 to help cover the cost of a second round of treatment for Celine.
Myotonic dystrophy is a genetic condition that leads to progressive muscle weakening. Symptoms can start at any age but they are generally more severe in children. There is currently no cure for the condition going forward. The couple has been told that their daughter will never have a normally functioning immune system. It is hoped that the stem cell therapy that Celine will receive in Mexico will slow the weakening of her muscles.
The treatment has not yet proved effective for muscular dystrophy, nor has it been approved by the NHS. As a result, the family had to raise £86,000 for the treatment. Stem cell treatment is a promising area of medicine, however, with the potential to significantly improve the lives of those with certain conditions
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