The family of a toddler is in a race against time to raise £250,000 for life-saving treatment after she was diagnosed with a condition so rare that only one other person in the UK has it. Three-year-old Dua Ahmad has been diagnosed with Spastic Paraplegia 50 (SP50), an exceptionally rare disease that causes paralysis starting from the toes and progressing to the brain. She is one of only two children in the country with this genetic condition, and without intervention, she faces quadriplegia by the age of 20.
Dua’s mum, Nadia, 38, has identified a medical trial in America for a drug named Elpida that uses gene therapy to potentially halt the progression of Dua’s symptoms. However, participation in the trial comes with a hefty price tag of £250,000, which Nadia will need to raise independently. Despite the daunting challenge, Nadia is determined to exhaust every option to secure the treatment for her daughter to ensure the best outcome possible.
Initially, Nadia noticed something was amiss with Dua when she was only a few weeks old. The toddler showed signs of lethargy and lack of responsiveness, prompting a series of medical visits that led to the SP50 diagnosis. The rarity of the condition left Nadia feeling devastated, as she had never encountered it before. With fewer than 100 documented cases worldwide, the family was faced with an overwhelming sense of uncertainty about Dua’s future.
The timeline for Dua’s condition is concerning, with the prospect of her needing a wheelchair by age ten and facing quadriplegia by age 20 if left untreated. Despite the challenges, Nadia remains optimistic about Dua’s progress and development, emphasising the importance of ongoing therapies to maintain flexibility and strength in her limbs. Dua’s recent milestone of learning to crawl at the age of three has been a significant achievement, showcasing her determination and resilience.
In the event that Dua is not selected for the American trial, the family will explore purchasing the medication once it gains approval, still requiring the substantial sum of £250,000. To aid in fundraising efforts, Nadia has set up a GoFundMe page to garner support from the community. The prospect of accessing this treatment could significantly impact Dua’s quality of life and overall prognosis, underlining the critical nature of securing the necessary funds for her care.