Kuwait first in Middle East to treat Duchenne muscular dystrophy

• Supporting the most vulnerable groups is a core principle of Kuwait’s foreign policy, said Abdulaziz Al-Jarallah, the Assistant Minister of Foreign Affairs for International Organisations and Minister Plenipotentiary.

Dr. Abdullah Al-Faras, Assistant Undersecretary of the Ministry of Health for Supporting Medical Services Affairs, announced that Kuwait is the first country in the Middle East to offer treatment for Duchenne muscular dystrophy, according to Al-Jarida newspaper.

Speaking on behalf of Dr. Ahmed Al-Awadi, the Minister of Health, at a celebration organized by the Centre for Genetic Diseases in cooperation with the Ministry of Foreign Affairs to mark World Duchenne Muscular Dystrophy Awareness Day, Al-Faras noted that the disease is a rare recessive genetic disorder linked to the X chromosome, affecting one in every 3,500 male births, and is one of the most common forms of muscular dystrophy.

Al-Faras confirmed the Ministry’s interest in registering the disease since 2000, with the Genetic Diseases Centre recording more than 130 cases of Duchenne muscular dystrophy (DMD) in Kuwaiti patients.

Deterioration of respiratory muscles

He explained that the disease used to claim lives in patients’ 20s and 30s due to the deterioration of respiratory muscles, but hope was renewed in 2016 when doctors deepened their understanding of the disease after identifying the genetic defect and its location in the human genome.

Dr. Abdullah Al-Faras stated that Kuwait is expanding the availability of genetic treatments as they are approved by the US Food and Drug Administration (FDA). The most recent advancement is gene therapy, which received FDA approval in June 2023, benefiting patients who meet the treatment criteria.

Integrated health and social care

He noted that Duchenne patients require comprehensive and integrated health and social care, which begins as soon as they are referred to the Genetic Diseases Centre, where a full genetic analysis is performed after obtaining approval from the Regulatory Committee for Gene Therapy, and the necessary treatment is prescribed.

He added that doctors provide care to patients through an integrated medical approach across all paediatricdepartments, in cooperation with neurologists, respiratory specialists, gastroenterologists, and physical therapists.

Kuwait has consistently sought to improve global health

For his part, Abdulaziz Al-Jarallah, the Assistant Minister of Foreign Affairs for International Organisations and Minister Plenipotentiary, stated in his speech that Kuwait, through its humanitarian diplomacy under the umbrella of its foreign policy, has consistently sought to improve global health, particularly regarding rare diseases, despite the absence of an official classification by the United Nations for those affected by such conditions.

He noted that, in addition to these efforts, Kuwait has taken on the responsibility of introducing a draft resolution to raise awareness of Duchenne muscular dystrophy (DMD) at the United Nations General Assembly, under the title of World Duchenne Muscular Disease Awareness Day.

He added that the resolution was adopted by consensus, with the Kuwaiti initiative receiving the support of 128 countries—the highest number during the 78th session of the United Nations General Assembly—resulting in the designation of September 7 each year as World DMD Awareness Day.

Community awareness

Al-Jarallah explained that the primary goal of organizing this event is to raise community awareness about the nature of this disease, as well as to provide support to individuals and families affected by it. He emphasized that supporting the most vulnerable groups is a core principle of Kuwait’s foreign policy.

He stated that establishing a World Day for DMD is a significant step toward generating the international momentum needed to combat the disease.

Al-Jarallah emphasized the ministry’s commitment to its prominent humanitarian role in promoting healthcare and providing assistance to the most vulnerable groups, particularly those with rare diseases, and its efforts to support them at both local and international levels. This aligns with its humanitarian vision and principles, which support health rights in accordance with the National Development Plan 2035.

He highlighted the ministry’s drive to create a healthy, inclusive environment through cooperation with relevant authorities and international organizations, ensuring patients receive necessary treatment and care. This also includes ensuring that national facilities can provide early diagnosis and secondary prevention for Duchenne muscular dystrophy and other rare diseases.

Building a brighter future

Al-Jarallah reaffirmed the ministry’s ongoing commitment to strengthening the international health system across all sectors, reflecting Kuwait’s dedication to building a brighter future and enhancing its global standing.

For her part, Dr. Laila Bastaki, head of the Centre, noted that Kuwait’s decision to propose September 7 as World Duchenne Muscular Disease Awareness Day at the United Nations was a milestone in serving those affected by the disease.

Supporting the dignity and rights of every child

She added that the decision was made to support the dignity and rights of every child with this condition and to foster a more compassionate and supportive world for individuals with this disease, their families, and the medical professionals confronting it. This decision underscores the need for global and international attention and emphasizes the importance of realizing our collective responsibility to protect and empower the most vulnerable groups affected by this disease.

She noted that Kuwait has adopted an approach aimed at achieving the Sustainable Development Goals by promoting health and well-being, as well as working to reduce inequality for those suffering from rare diseases. This approach integrates health, education, economic stability, equality, and sustainable community development.

She announced that the National Registry of Neuromuscular Diseases has recorded approximately 141 cases of Duchenne muscular dystrophy of varying ages since its inception.

She explained that muscle atrophy in Duchenne muscular dystrophy results from a genetic defect. However, in some rare cases, it may occur suddenly without a genetic origin, presenting as weakness and atrophy with ongoing effects on the body’s muscle health.

Dr. Laila Bastaki explained that individuals with this disease face various motor difficulties, especially when trying to walk or climb stairs. Over time, the patient’s condition worsens, leading to motor disabilities and numerous health complications in some organs, particularly the heart muscle, respiratory muscles, and the spine.

She noted that the ministry has adopted and provided drug treatment for the disease since 2016, despite its high cost. Kuwait was one of the first countries in the region to offer this treatment immediately after its approval by specialized bodies. Additionally, the ministry has adopted the promising gene therapy “Evides,” which is administered in a single dose and has international approval.

She emphasized that priority has been given to children aged 4-6 years with Duchenne muscular dystrophy in the country, allowing them to receive treatment despite the high cost, which approaches three million dollars. This reflects Kuwait’s renewed and pioneering commitment to providing innovative, safe, and effective treatments for rare genetic diseases and enhancing the quality of life for those affected.

Duchenne muscular dystrophy’s early detection

Bastaki added that the center offers an integrated health service through a multidisciplinary medical team specializing in muscular dystrophy. This team is responsible for diagnosis and early detection through precise genetic testing, as well as providing genetic counseling and traditional and modern gene therapy.

She also highlighted that the center provides safe reproductive solutions to prevent the birth of children with this disease. This includes pre-implantation examination of fertilized eggs and various diagnostic methods during pregnancy and after childbirth to detect symptoms early in vulnerable families.