Doha Dr. Ahmed Al Hammadi, Executive Director of Pediatrics at Sidra Medicine in the State of Qatar, revealed that his country has provided treatment for Duchenne muscular dystrophy patients free of charge to its citizens, explaining that the value of this treatment amounts to about 10 million Qatari riyals (the dollar is 3.65 riyals).
The Executive Director of Pediatrics at Sidra Medicine, in an interview with Al Jazeera Net, confirmed that the importance of this new treatment lies in completely changing the patient’s condition from general deterioration to stability and growth normally again, pointing out that the State of Qatar is the first in the region to implement this treatment. Treatment through Sidra Medicine.
He pointed out that providing such modern and advanced treatments gives hope to many people suffering from this incurable disease, noting that Sidra Medicine is a trusted partner with extensive experience in the field of health care that contributes to consolidating Qatar’s position as the best choice for caring for children who suffer from hereditary diseases. Complex and rare.
Below is the text of the dialogue:
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In the beginning…what is Duchenne muscular dystrophy?
Duchenne muscular dystrophy is known as a genetic disorder that gradually weakens the body’s muscles. The reason for its occurrence is due to the presence of incorrect or missing information or genetic sequence that prevents the body from making the proteins necessary to build and maintain healthy muscles. This disease usually spreads more among males and affects about one boy out of every boy. 3500 children.
As for girls, they carry the gene responsible for the disease, but the symptoms do not appear on them. Usually, children with this type of disease usually live for 20 years, before the discovery of a new treatment, which will undoubtedly have a significant impact on increasing the lifespan of affected children and even improving their quality of life.
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How is the condition detected or diagnosed?
There are a number of symptoms that appear in the child when he reaches the age of two, as he notices delayed growth, muscle weakness, falling while walking or inability to walk, frequent falls, difficulty getting up from a lying or sitting position, walking on his toes, muscle pain and stiffness, learning difficulties, and a decline in physical function. Developing motor skills. If these symptoms appear, parents must go to the doctor immediately for diagnosis and treatment.
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What is the condition of a patient with this disease and what is the current treatment method?
The affected child usually lives for a few years and dies at an early age as a result of infection with this disease, which leads to deterioration in the functions and muscles of the body. However, recently a new gene therapy has appeared that contributes to stopping the deterioration of the condition. It is a single needle, as this innovative medicine works to replace The damaged gene responsible for the disease is replaced by a healthy gene, slowing the progression of the disease and enhancing muscle function.
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How are patients selected for treatment? What is the cost of this treatment?
Patients are selected based on a number of criteria, including that the child must be no more than 4 years old and suffer from Duchenne muscular dystrophy based on a genetic medical diagnosis that actually determines the presence of the disease, and that the patient must not have undergone or received any previous genetic treatments.
The criteria also include that the patient does not suffer from a deficiency in exon 8 or exon 9, which are parts of the Duchenne gene that is examined to confirm the criteria for conducting the treatment. The value of this treatment is about 10 million Qatari riyals and is provided to Qatari patients free of charge. It can also be provided to non-Muslims. Citizens through charitable organizations.
We became the first hospital outside the United States to offer gene therapy to pediatric Duchenne muscular dystrophy patients over six years of age.
For more details about our Duchenne Muscular Dystrophy treatment program, please visit our website https://t.co/7tHhKEL1YS or call 3333… pic.twitter.com/B5ESTd9Aqx
— Sidra Medicine (@SidraMedicine) October 22, 2024
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Has this treatment been implemented and what is its impact on improving the quality of life of patients and their families?
7 cases were treated with the new genetic drug, including 5 Qatari citizens and two international patients, and the results demonstrated a very noticeable improvement for the patients, who began to regain much of their vitality and activity shortly after treatment.
There is no doubt that the launch of this treatment is an important step towards improving the quality of life of Duchenne muscular dystrophy patients and their families, as it gives them hope for a better future and opens new horizons for the treatment of rare genetic diseases. Through this treatment, Sidra Medicine confirms its leadership in the field of health care and its provision of innovative treatments that contribute to improving lives. Patients.
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What about the side effects of the new gene therapy?
Side effects reported during clinical studies include an allergic reaction to the components of the treatment, an increase in liver function, nausea, and vomiting while taking the treatment. Such advanced gene therapies are only used inside hospitals and under complete medical supervision and under the supervision of a medical team specialized in gene therapy to deal immediately with… No side effects.
At Sidra Medicine, all children who were given Elephdis treatment responded well to the treatment with no reported side effects, and the medical teams at our center are in constant contact with families to follow up on their children’s post-treatment care in our specialized clinics.
Baby Chris Cake is the first patient at our hospital to receive pioneering gene therapy for Duchenne muscular dystrophy. ️
This innovative treatment, called ELIVIDIS, gives children with Duchenne muscular dystrophy the hope of living a normal life.
In this video, our staff and doctors shed more light on this treatment… pic.twitter.com/pckOioiKeF
— Sidra Medicine (@SidraMedicine) July 23, 2024
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Is this new treatment the ultimate treatment for Duchenne muscular dystrophy, or are there other studies in this regard?
There are many clinical trials currently active to treat Duchenne muscular dystrophy, the most important of which is restoring or replacing the dystrophin gene, which is the missing or defective protein in Duchenne muscular dystrophy. There are also some clinical trials focusing on drugs that can protect muscle cells even if they do not treat the genetic defect of the disease, and finally There are clinical trials aimed at improving muscle function, such as increasing muscle growth or reducing muscle inflammation.
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What criteria does the US Food and Drug Administration rely on to approve such treatments?
The US Food and Drug Administration (FDA) relies on a set of strict standards to approve gene therapies, including the revolutionary gene therapy “Elividis” for patients with Duchenne muscular dystrophy. These standards include proven effectiveness. The treatment must prove its effectiveness in improving patients’ condition and reducing the progression of the disease. In addition to safety, the side effects of treatment must be acceptable compared to the benefits. Standards also include quality. The treatment must be manufactured according to strict standards to ensure its quality and safety.
Before any gene therapy enters clinical trials, it undergoes tests in laboratories and on animal models to evaluate its effectiveness and safety. These pre-clinical studies are considered important in determining the appropriate dose, knowing the potential side effects, and evaluating the treatment period and its ability to achieve the desired effect.
After conducting studies, initial approval is taken to begin clinical trials, which pass through 3 clinical stages until all evidence and data are reviewed to determine the safety and effectiveness of the genetic drug and other medical drugs.
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In the end…what is your message to patients and their families?
My message to them is that there is always hope in light of scientific and medical progress to treat such difficult diseases. Adopting such gene therapies gives hope to patients with rare and complex diseases who often do not have treatment options to stop the effects of the disease or its complications.
Sidra Medicine works to change the lives of children with Duchenne muscular dystrophy in Qatar and the Arab world. We are proud of the great trust our center enjoys and the high skills and experience enjoyed by our medical teams. Our services are distinguished and world-class, and we remain committed to providing the highest standards of health care and the latest medical treatments to all our patients.
Providing such modern and advanced treatments confirms that Sidra Medicine is a trusted partner with extensive experience in the field of health care, and these medical efforts contribute to consolidating Qatar’s position as the best choice for caring for children who suffer from complex and rare genetic diseases.