Campaign for Yakup: Providing Hope for a Better Future

 

When we discovered his illness, he was  just six months old. Since the age of six months, he has struggled with hospitals, tests, physical therapy, night splints, running tests, walking assessments, and medications, my dear son, Yakup. Today, we are starting a campaign for you. Years ago, my cousin suffered from this illness, and I knew how challenging life was for him until he passed away at the age of 16. Back then, there was no treatment available. Now, my son has the same condition, and there is treatment. I have never lost hope that he will one day heal, and I won't lose it. I kindly request your help to assist my son in obtaining the necessary medication for his treatment.

 

 

 

 

 

Duchenne Muscular Dystrophy (DMD) is a genetic disease caused by a mutation in the dystrophin gene found on the Xp21 chromosome. Mutations in the dystrophin gene lead to progressive muscle fiber degeneration and weakness. This weakness may initially manifest as difficulty in walking but progressively worsens, eventually making it impossible for affected individuals to perform daily life activities, leading to the need for a wheelchair. Cardiac and orthopedic complications are common, and death usually occurs in the twenties due to weakness in respiratory muscles or cardiomyopathy.

 

 

 

In July, the FDA granted accelerated approval to ELEVIDYS (delandistrogene moxeparvovec-rokl), a micro-dystrophin gene therapy used to treat individuals with Duchenne muscular dystrophy aged 4-5.The approval of ELEVIDYS marks a turning point in Duchenne treatment. ELEVIDYS is the first and only gene therapy approved for Duchenne. ELEVIDYS addresses the underlying genetic cause of Duchenne by providing a shortened form of dystrophin to muscle cells, compensating for the mutation causing a lack of dystrophin protein in the dystrophin gene. This accelerated approval is based on an increase in micro-dystrophin protein expression in skeletal muscle. ELEVIDYS is supported by efficacy data from two clinical studies: SRP-9001-102 and SRP-9001-103, along with safety data from SRP-9001-101, SRP-9001-102, and SRP-9001-103.

 

ELEVIDYS currently treats Duchenne patients aged 4-5 who can walk and do not have antibodies against AAVrh74. Currently, Yakup is 5 years old and meets these criteria. HOWEVER, OUR INSURANCE COMPANY, OSCAR, DOES NOT COVER THE MEDICATION. Our insurance company stated that those who take the drug did not show significant improvement compared to the placebo and cited the small number of individuals in the clinical trial, denying coverage twice. But I do not believe this. We have children who, even at the age of 11, after taking ELEVIDYS, can walk, run, and jump. The most crucial thing to know is that these children start using a wheelchair at no later than 12 years old. The effectiveness of the medication is clear. Yakup will turn 6 in March and NEEDS TO RECEIVE THIS MEDICATION WITHIN 4 MONTHS. Support us and help us provide Yakup with a better future.