Yunus Kidwai will turn six in September, but the anticipation of his birthday is marred with a sense of dread for his parents. Their child suffers from a rare and genetic disorder known as Duchenne muscular dystrophy (DMD), a condition that progressively worsens over time, leaving those affected in a debilitating and often fatal condition.
Yunus’s parents are racing against time to secure Elevidy, a vital gene therapy. However, this one-time treatment comes with a crucial condition: it must commence before Yunus’s sixth birthday to be effective. Once this day passes, the window of opportunity slams shut, allowing the disease to continue its relentless march.
At present, Al Jalila Children’s Specialty Hospital in Dubai stands as a lifeline for families contending with DMD outside the United States. Yunus’s parents, who have travelled from India, are desperately seeking this critical treatment for their only son.
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Despite Yunus’s current ability to walk and run, the ominous threat of muscular degeneration looms over his future. Climbing steps is already challenging, and in the years ahead, his muscles will weaken to the point where standing or maintaining balance becomes impossible. By his teenage years, Yunus could find himself confined to a wheelchair, with his arms, lower legs, and trunk succumbing to muscle weakness.
Reaching his 25th birthday would be a stroke of luck for Yunus, considering many with DMD don’t make it that far.
Adding to the family’s woes is the huge cost of Elevidy – a hefty Dh11.75 million. Yunus’s parents say they simply can’t afford this overwhelming amount.
The child’s mother, Kulsum Kidwai, grimly acknowledges their distress saying: “The gene therapy can give him a happy life because Yunus is a very happy soul. He loves to colour, paint, and recite rhymes,” she says.
Fazal Yasin Kidwai, Yunus’s father and a medical representative in India, reflected on the impact August 2023 had on their once-happy family. He shared, “It was a tough realisation when we noticed Yunus having difficulties and took him to the local doctor. The diagnosis revealed that he has DMD. Initially, we didn’t even know what it was, but when we found out, our hearts sank.”
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Seeking confirmation, he took the child to the All India Institute of Medical Sciences (AIIMS) in New Delhi, where subsequent tests confirmed the disease.
“We are devastated and just could not bear the thought of seeing our child wither away,” he said.
Fazal said the family rushed to Dubai after doctors at AIIMS informed them that the gene therapy is available only at the city’s Al Jalila Speciality Hospital and Medcare Women & Children Hospita aside from the United States.
“Yunus was examined by doctors at Al Jalila earlier this week, and they conveyed that the gene therapy is our last hope,” said Fazal, displaying hospital papers.
A comment from Al Jalila Specialty Hospital was not available.
In June last year, the US biotech firm Sarepta Therapeutics gained approval for the $3.2 million Elevidys therapy for the treatment of paediatric patients aged 4 through 5 with Duchenne muscular dystrophy. The gene therapy at Nemours Children’s Hospital in Orlando has already changed a young boy’s life, with Dr. Omer Abdul Hamid, a pediatric neurologist at the facility, describing the treatment as a game-changer for families and communities.
Last year, Sarepta Therapeutics paused a clinical trial for its second-generation Duchenne muscular dystrophy medicine due to a safety incident. However, on Tuesday, the company announced positive Phase II results after resuming the halted trial.
How the drug works
The gene transfer therapy employs an adeno-associated virus vector to transport an engineered mini-dystrophin gene into muscle cells, facilitating the production of dystrophin—a crucial protein that aids in maintaining the cohesion of muscle cells. The drug is administered through a one-time intravenous infusion.
What causes DMD
Sometimes shortened to DMD or Duchenne, this rare disease stems from a genetic mutation inhibiting the production of the protein dystrophin, crucial for muscle contraction’s shock-absorbing function. Without dystrophin, muscles progressively weaken, lose repair ability, and lead to walking and breathing difficulties. Ultimately, Duchenne becomes irreversible and fatal, impacting the heart and respiratory muscles. Despite advancements in management, there is currently no cure. Primarily affecting males, with 1 in 3,500 to 5,000 boys born worldwide having Duchenne, it can also rarely affect females.