Alyssa's fight: Parents launch campaign for urgent treatment of rare condition

IOL News

6 days ago

Somerset Park couple raises funds for daughter diagnosed with MPS I.
Image: Supplied
A young couple from Somerset Park, KwaZulu-Natal, has launched a desperate effort to secure life-saving treatment for their infant daughter, Alyssa Cas Aldworth, after she was diagnosed with a rare and potentially fatal genetic disorder.
Alyssa, born on September 28, 2024, was recently diagnosed with Mucopolysaccharidosis Type 1 (MPS I), a rare condition caused by the absence of the IDUA gene.
The gene is responsible for producing an enzyme that breaks down complex sugars in the body. Without treatment, children with MPS I rarely live past the age of ten.
Her father, 29-year-old Trysten Aldworth, described the news as devastating. "Hearing that your only daughter has a rare genetic disorder with an extremely high mortality rate has broken us beyond words," he said. "It's something no parent should ever have to hear."
Alyssa, affectionately known as Ally, spent her first 10 days in a neonatal intensive care unit due to severe heart and lung complications. Despite initial health setbacks, she has continued to show signs of improvement with regular medical monitoring.
The condition requires immediate medical intervention in the form of Enzyme Replacement Therapy (ERT), using a drug called laronidase (Aldurazyme). However, the therapy comes at a steep cost, quoted at R78,064.64 per month, excluding VAT and must be administered weekly for life.
Even with ERT, the enzyme cannot cross the blood-brain barrier, limiting its effectiveness in halting neurological damage caused by MPS I. As a result, doctors are urgently considering a Hematopoietic Stem Cell Transplantation (HSCT), a more aggressive treatment aimed at halting disease progression if performed within the first two years of life.
The cost of HSCT abroad is estimated between £150,000 and £200,000 (approximately R3.6 million to R4.9 million), though the family hopes to explore a more affordable option within South Africa. A consultation with a haematologist is scheduled this week to assess the feasibility of the treatment locally.
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The couple launched a campaign on Back a Buddy with the aim to raise R468,000 and have raised close to R206,000.
Aldworth who has been married to his wife Jade for five years, said their community's support has been overwhelming. "In just days, the amount of people who have called, messaged, donated or shared Ally's story has been nothing short of amazing. It's scary, but in the best possible way."
Ally's middle name, Cas, honours Jade's late father, Craig Andrew Suttie, who died in July 2023 from cerebral malaria at the age of 47. The family, together since 2013, remains hopeful as they navigate complex medical decisions.
"We just want what every parent wants; for our child to be healthy, to live a full life, and one day walk down the aisle," Trysten added. "We're going to do everything in our power to make that possible."
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