Racing Against DMD: Yunus's Elevidy Hope
**Title: A Family's Race Against Time: Seeking Hope for a Son Battling Duchenne Muscular Dystrophy**
*Subheadings:*
1. *The Harsh Reality:* Yunus's Struggle with Duchenne Muscular Dystrophy*
2. *Critical Window:* The Urgency of Elevidy Treatment Before Yunus's 6th Birthday*
3. *Desperate Journey:* Yunus's Parents Travel from India to Dubai in Search of Lifesaving Therapy*
4. *Financial Burden:* The Daunting Cost of Elevidy and the Family's Plea for Assistance*
5. *A Happy Soul in Distress:* Kulsum Kidwai's Heartbreaking Acknowledgment of Yunus's Condition*
6. *A Tough Realization:* Fazal Yasin Kidwai Reflects on Discovering Yunus's Diagnosis and the Family's Devastation*
7. *Seeking Hope at Al Jalila Specialty Hospital:* The Last Resort for Gene Therapy*
8. *The Game-Changing Elevidys Therapy:* A Look at the Positive Impact on Other Patients*
9. *Resuming Hope:* Sarepta Therapeutics' Positive Phase II Results After a Safety Incident*
10. *Understanding Duchenne Muscular Dystrophy:* Causes, Impact, and the Pursuit of a Cure*
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Yunus Kidwai's upcoming sixth birthday should be a time of celebration, but for his parents, it marks a race against time. Their son is battling Duchenne muscular dystrophy (DMD), a rare genetic disorder that progressively debilitates those affected. The family's hope lies in securing Elevidy, a gene therapy that must commence before Yunus turns six to be effective.
The struggle intensifies as Yunus's parents, who have traveled from India, seek this vital treatment at Al Jalila Children's Specialty Hospital in Dubai, the lifeline for families grappling with DMD outside the United States. With a hefty cost of Dh11.75 million for Elevidy, the family faces a financial burden that adds to the distress of their son's condition.
Yunus, currently able to walk and run, faces an uncertain future as muscular degeneration threatens to take away these abilities. Climbing steps has become challenging, foreshadowing a time when standing or maintaining balance will be impossible. The grim reality is that many with DMD don't reach their 25th birthday.
Despite the financial strain, Yunus's mother, Kulsum Kidwai, holds on to hope, recognizing that Elevidy could offer a chance at a happy life for her son. Fazal Yasin Kidwai, Yunus's father and a medical representative in India, reflects on the tough realization of discovering Yunus's diagnosis, and the family's subsequent rush to Dubai in pursuit of the gene therapy available at Al Jalila Specialty Hospital.
Elevidy, a one-time treatment, gained approval from the US biotech firm Sarepta Therapeutics for pediatric patients aged 4 through 5 with DMD. The gene therapy utilizes an adeno-associated virus vector to transport an engineered mini-dystrophin gene into muscle cells, facilitating the production of dystrophin—a crucial protein for muscle cohesion. Administered through a one-time intravenous infusion, Elevidy has shown promising results, prompting a positive Phase II outcome after a temporary clinical trial pause.
While Elevidy offers hope, the reality is that DMD, caused by a genetic mutation inhibiting dystrophin production, remains incurable. The disease primarily affects males, with worldwide incidence at 1 in 3,500 to 5,000 boys born, and rarely impacting females.
As Yunus's family grapples with the challenges posed by DMD and the urgency for Elevidy, their story becomes a poignant reminder of the relentless pursuit of hope in the face of a devastating and rare genetic disorder.
By: Sahiba Suri
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