Duchenne Muscular Dystrophy (DMD) and Elevidys Gene Therapy: Hope for Children
Duchenne Muscular Dystrophy (DMD) is a genetic muscle disease that occurs in childhood. This disease causes the muscles to weaken and waste away, seriously affecting the quality of life of children. Children struggling with DMD, Elevidys Access to innovative treatments such as gene therapy is awaiting access. This treatment offers great hope for families as it has the potential to stop or slow the progression of the disease.
What is Elevidys Gene Therapy?
Elevidys is a gene therapy method developed for DMD disease. This treatment aims to correct the genetic defects caused by the disease. Elevidys is applied directly to the muscle cells of patients, helping the muscles to regain strength. Clinical trials The data obtained as a result of the study show that this treatment is effective. However, there are some obstacles to accessing this treatment in Turkey.
The Situation of Families Struggling with DMD in Turkey
DMD Türkiye Association Izmir Provincial Representative Magnolia Alay, made important statements on this subject. Alay, his son Cinar AlpHe stated that he participated in the clinical trials of Elevidys at the age of five. Çınar is currently in very good shape compared to his peers. He can climb stairs in his daily life and walk to school. This is considered an almost impossible feat for other children with DMD.
Cost and Access Issues of Elevidys
The fact that Elevidys treatment is not under state guarantee in Turkey poses a major problem for families. Alay states that the cost of individual purchase of the drug is 3.2 million dollars. There are approximately 5 thousand children with DMD in Turkey. This situation makes it very difficult for families to access treatment. It is believed that the price of the drug will decrease in case of a bulk purchase.
The Role of the State and Social Policy
The DMD Turkey Association emphasizes that savings in health are unacceptable. Alay emphasizes that the state must take urgent steps in this regard by saying, “Our children do not have time to wait.” Families fighting for the right to life of children with DMD have organized protests in front of the ministry in the cold winter conditions of Ankara. However, no concrete steps have been taken as a result of these protests.
Community Sensitivity and Need for Support
The biggest expectation of families struggling with DMD is that society becomes more sensitive to this issue. Increasing awareness about the disease will be an important step in accessing treatment. Families need support not only from the state but also from society. Campaigns to be carried out on this issue and awareness activities to be carried out on social media will provide a great opportunity for patients to have their voices heard.
International Support and Collaborations
It is known that Elevidys treatment is provided free of charge through insurance companies in the US and some foundations in Russia. Establishing similar collaborations in Turkey could facilitate access to treatment for children with DMD. Such international support is critical to improving the quality of life of patients.
In Conclusion: Looking to the Future with Hope
Duchenne Muscular Dystrophy is not just a disease, it is also a condition that deeply affects the lives of families. The state guarantee of innovative treatments such as Elevidys in Turkey will be a great gain for children struggling with this disease. The hope of families lies in the steps that society and the state will take in this regard. The fight for the right to life of children struggling with DMD disease must continue.
