Gene Therapy in the UAE

In a relatively short period, the UAE has become one of the notable centers of advanced medicine in the region, actively developing fields in genomics, precision medicine, and innovative therapy for rare diseases. Today, gene therapy programs are available in the country for patients with Duchenne muscular dystrophy, spinal muscular atrophy, sickle cell anemia, and transfusion-dependent beta-thalassemia, which is confirmed by successful statistics and official reports from profile regulators and healthcare authorities.

The healthcare system in the UAE operates within a framework of state regulatory supervision. At the federal level, the Emirates Drug Establishment oversees the approval and circulation of innovative medical products, while in the Emirate of Abu Dhabi, healthcare activities are regulated by the Department of Health – Abu Dhabi (DoH). All regulation and control processes are aimed at ensuring the quality, safety, and compliance of medical services and products with established requirements.

In UAE medical centers offering gene therapy, patients are supported by international teams of doctors, nurses, and administrative specialists who speak Arabic, English, Russian, Hindi, and other languages. This multilingual environment makes communication more comfortable for patients from different countries and helps minimize language barriers when discussing complex genetic treatment protocols.

Advanced methods and gene therapy products have been introduced into practice in the UAE, for example, CASGEVY – for sickle cell anemia and beta-thalassemia (therapy using CRISPR/Cas9 genome editing technology). Or, ELEVIDYS — a gene therapy product to treat patients with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene.

The UAE is one of the most convenient transport hubs between Europe, Asia, and Africa, making the country an ideal destination for international patients who value flights with a minimum number of transfers and a developed infrastructure for providing medical services.

Patients undergoing gene therapy are provided with comfortable conditions throughout their treatment journey — both during hospital admission and outpatient care, with accommodation arranged in well-appointed hotels or apartments.

Spinal muscular atrophy (SMA) is a rare hereditary neuromuscular disease in which motor neurons responsible for transmitting signals from the brain and spinal cord to the muscles are gradually affected. As a result, muscles weaken, lose volume, and gradually stop performing their functions. SMA is a serious genetic disease that requires early diagnosis, precise genetic confirmation, and a correctly structured treatment strategy.

Today, the UAE is becoming one of the promising destinations for the treatment of SMA in children, especially for families who value access to advanced medical technologies, modern management protocols, and an international level of medical infrastructure.

In spinal muscular atrophy, the function of the SMN1 gene, which is necessary for the production of a protein that supports the vital activity of motor neurons, is disrupted. When there is not enough of this protein, motor neurons gradually degenerate, and muscles stop receiving full signals to contract. This leads to muscle weakness, decreased motor activity, impaired motor development, and in severe forms, to problems with swallowing and breathing.

SMA can manifest in different ways: from severe forms beginning in the first months of life to more slowly progressing variants identified later in children or already in adulthood. The earlier symptoms appear, the higher the risk of rapid disease progression.

The clinical manifestations of SMA depend on the type of disease and the age at which symptoms begin; however, the most common signs are significant muscle weakness, delayed motor development, loss or decrease in previously acquired skills, difficulty sitting up independently, standing and walking, frequent falls, hand tremors, weakness of the trunk muscles, and in some patients, problems with swallowing and respiratory function.

For many families, the deciding factor is precisely the early detection of SMA, as modern therapy methods are most effective when treatment starts as early as possible: medical capabilities in the UAE today open access to innovative methods — the disease is treatable with gene therapy.

A diagnosis of spinal muscular atrophy is confirmed primarily by genetic testing, which allows for the identification of a mutation or deletion in the SMN1 gene. Additionally, doctors evaluate the clinical picture, neurological status, respiratory function, nutritional status, and the child’s overall motor development. In some cases, the examination program may include electromyography, functional assessment, and consultations with a geneticist, pediatric neurologist, pulmonologist, and rehabilitologist.

For international patients, it is particularly important that the diagnosis be comprehensive. It is this approach that allows for the correct determination of the SMA type, assessment of disease severity, clarification of indications for therapy, and selection of the optimal treatment route.

In recent years, the approach to treating spinal muscular atrophy has changed dramatically. While previously assistance to patients was limited mainly to supportive therapy, today drugs that affect the biological mechanism of the disease are available worldwide. One of the first such drugs was SPINRAZA® (nusinersen), approved by the FDA in December 2016 for the treatment of SMA in pediatric and adult patients.

Another drug – Zolgensma® (Onasemnogene abeparvovec-xioi): is a one-time gene therapy aimed at eliminating the root cause of the disease by replacing the defective SMN1 gene.

These therapy methods provide new hope and contribute to improving the quality of life for children and families facing SMA.

In addition to medical treatment, rehabilitation programs are of great importance and yield positive results. Goals of rehabilitation:

• Preservation of the current state and slowing of deterioration
• Increasing or maintaining the child’s independence
• Prevention of joint deformities
• Maintenance of muscle mass
• Fighting respiratory complications
• Providing social adaptation and quality of life

It is important to understand that a specific treatment option for SMA is always selected individually. It depends on the patient’s age, genetic confirmation of the diagnosis, the clinical type of the disease, the state of the respiratory system, motor status, the timing of symptom onset, and criteria for admission to a particular therapy. This is why SMA treatment should be built not around a single drug, but around the patient’s full clinical route.

The UAE attracts international patients with a combination of several factors: modern medical infrastructure, active development of genomic and precision medicine, state support for rare disease programs, a multilingual environment, convenient international logistics, and the ability to organize treatment in a comprehensive support format.

In SMA, a multidisciplinary approach is particularly important. The treatment and monitoring of a child usually require the participation of a pediatric neurologist, a geneticist, intensive care specialists, a pulmonologist, a nutritionist, a rehabilitologist, and long-term monitoring coordinators. It is this format that allows not only for prescribing therapy but also for ensuring full patient management before and after treatment.

Duchenne muscular dystrophy (DMD) is a severe hereditary disease in which muscle weakness gradually increases, motor activity decreases, and over time, the respiratory and cardiovascular systems may become involved. The disease is associated with a mutation in the DMD gene, which prevents the body from producing sufficient amounts of dystrophin — a protein necessary for normal muscle function. The disease occurs predominantly in boys and belongs to rare but clinically significant hereditary pathologies.

Today, the UAE is becoming one of the promising destinations for families seeking modern treatment options for DMD in children. Since 2024, a gene therapy program for DMD has been introduced in the UAE, which was previously only available in the USA.

Duchenne muscular dystrophy (DMD) is a severe hereditary disease in which muscle weakness gradually increases, motor activity decreases, and over time, the respiratory and cardiovascular systems may become involved. The disease is associated with a mutation in the DMD gene, which prevents the body from producing sufficient amounts of dystrophin — a protein necessary for normal muscle function. The disease occurs predominantly in boys and belongs to rare but clinically significant hereditary pathologies.

Today, the UAE is becoming one of the promising destinations for families seeking modern treatment options for DMD in children. Since 2024, a gene therapy program for DMD has been introduced in the UAE, which was previously only available in the USA.

In DMD, muscles gradually lose the ability to function normally because, without dystrophin, muscle fibers become vulnerable to damage and are eventually replaced by fatty and connective tissue. The first signs of the disease often appear in early childhood: it becomes harder for the child to run, climb stairs, get up from the floor, and they fall more often, with a “waddling” gait appearing. As the disease progresses, weakness in the leg muscles and trunk increases, followed by potential issues with breathing and heart function.

For children suspected of having DMD, it is especially important to undergo genetic diagnosis as early as possible and receive consultation from profile specialists. Early diagnosis allows for a faster assessment of the mutation type, determination of available therapy options, and timely start of treatment, monitoring, and rehabilitation. In such diseases, time is of critical importance because early intervention helps to maintain motor function and the child’s quality of life for longer.

Parents should seek consultation if the child exhibits:

• frequent falls and clumsy gait
• difficulty climbing stairs or getting up from the floor
• pronounced muscle weakness in the legs
• walking on toes
• enlarged calf muscles
• reduced endurance compared to peers

Even if symptoms seem nonspecific, it is important not to delay examination if a hereditary muscle disease is suspected.

Diagnosis is usually built on a combination of clinical assessment, genetic testing, and additional studies necessary to evaluate the child’s overall condition and the stage of the disease. To plan treatment, it is important to confirm the mutation in the DMD gene, assess motor function, the state of the respiratory system, heart, and overall rehabilitation potential.

The approach to treating Duchenne muscular dystrophy is changing worldwide: in addition to standard supportive therapy, innovative methods aimed at the biological cause of the disease are becoming increasingly important. One such direction has become ELEVIDYS gene therapy; however, the decision on therapy requires a very careful selection of the patient and observation in a specialized center.

In Duchenne muscular dystrophy, muscles are gradually damaged due to a deficiency of dystrophin, which leads to reduced muscle strength and loss of motor functions. Therefore, treatment aimed at increasing dystrophin production is considered an important part of the early disease therapy strategy. ELEVIDYS is designed to help the body produce a new, shortened form of the protein — ELEVIDYS micro-dystrophin. This protein is smaller than natural dystrophin but retains key functional segments necessary for the formation of a working protein.

The therapy is based on two important components:

• ELEVIDYS vector — a delivery system that transfers therapeutic genetic material into muscle cells
• ELEVIDYS transgene — a genetic instruction that tells the body how to synthesize the new form of the protein micro-dystrophin

The gene therapy procedure with ELEVIDYS takes place in several stages:

1. Confirmation of treatment indications
   The first stage is determining whether ELEVIDYS therapy is suitable for the patient. For this, the doctor prescribes necessary examinations, including genetic and laboratory analyses. They help confirm the diagnosis, clarify the mutation type, and evaluate whether the child meets the criteria for the therapy.
2. Preparation for treatment
   If the patient is recognized as a suitable candidate, the preparatory stage begins. Before the infusion, additional examinations are conducted to record baseline values for subsequent comparison after therapy. Also, the family receives detailed explanations about concomitant treatment, including mandatory oral corticosteroid intake before and after infusion, as this is part of the safety protocol.
3. Day of therapy
   ELEVIDYS is administered once intravenously. The infusion itself usually takes about 1–2 hours; however, typically, the patient spends the whole day in the medical center as observation by the medical team is required before and after administration.
4. Post-treatment observation
   After therapy, it is particularly important to strictly follow the subsequent observation plan. Usually, families of international patients need to stay in the country where the treatment took place for at least 2-3 months after gene therapy and undergo weekly monitoring; however, exact routing and timing depend on the condition of the specific patient and are determined individually by the specialist according to the treatment plan. During this period, laboratory indicators, primarily liver function, as well as the child’s general condition and therapy tolerance, are monitored

The UAE attracts international patients with a combination of several factors: modern medical infrastructure, active development of genetic medicine, convenient logistics, a high level of service, and the ability to organize treatment in a comprehensive support format. For families considering Duchenne muscular dystrophy treatment in the UAE, not only technology is important, but also a clear patient journey — from initial document review to consultation, clinic selection, trip organization, and long-term observation.

Modern possibilities for treating rare hereditary blood diseases in the UAE offer completely new treatment methods, making the UAE one of the most promising destinations for treating international patients with beta-thalassemia and sickle cell anemia.

The development of genetic medicine, cellular technologies, and specialized centers in the country opens access to modern therapy methods, one of which is CASGEVY® — the first approved CRISPR/Cas9 therapy for both of these diseases.

Beta-thalassemia is a hereditary blood disease in which the body does not produce enough normal hemoglobin. Hemoglobin is needed by red blood cells to transport oxygen, so severe forms of the disease lead to significant anemia, weakness, growth retardation, enlargement of the liver and spleen, skeletal changes, and the need for regular blood transfusions. The most severe form is transfusion-dependent beta-thalassemia, in which patients require permanent transfusions.

In children, severe beta-thalassemia usually manifests as early as the first months or first year of life. Pallor, jaundice, fatigue, shortness of breath, physical developmental delay, bone deformities, and abdominal enlargement due to hepatosplenomegaly are possible.

Sickle cell anemia belongs to a group of hereditary hemoglobin diseases in which red blood cells become stiff and acquire a characteristic sickle shape. Such cells pass through vessels poorly, can block blood flow, and cause pain crises, anemia, infections, and gradual organ damage. Symptoms often begin in early childhood.

For patients with sickle cell disease, the following are particularly characteristic:

• pain crises
• anemia and weakness
• jaundice
• increased susceptibility to infections
• risk of damage to bones, lungs, spleen, and other organs

Traditionally, treatment included blood transfusions, chelating therapy to remove excess iron, prevention of complications, observation by a hematologist, and, in some cases, hematopoietic stem cell transplantation. Bone marrow transplantation is considered a potentially curative treatment method, but today new gene-cell approaches have appeared for some patients.

CASGEVY® (exagamglogene autotemcel) is a one-time therapy based on CRISPR/Cas9 genome editing. It is approved:

• for patients 12 years and older with sickle cell disease and recurrent vaso-occlusive crises
• for patients 12 years and older with transfusion-dependent beta-thalassemia

The meaning of the therapy is to increase the production of fetal hemoglobin (HbF). For this, the patient’s own blood stem cells are taken, edited in a laboratory, and then returned after a preparatory treatment stage. Increasing HbF helps to reduce red blood cell abnormalities in sickle cell anemia and reduce or eliminate the need for regular transfusions in transfusion-dependent beta-thalassemia.

For international patients, this means access to:

• modern hematological and genetic diagnosis
• a multidisciplinary team of specialists
• high-tech cellular and gene therapy programs
• an organized treatment route and subsequent observation

At the same time, it is important to understand that CASGEVY is not suitable for all patients.

The decision on the possibility of treatment is made only after a full examination, assessment of age, clinical severity, indications, risk of complications, and compliance with approved criteria.

It should be noted that in addition to the already available therapy methods, other innovative approaches to treating beta-thalassemia and sickle cell anemia continue to develop worldwide, including clinical trial programs. However, the final decision on indications, acceptability of therapy, and possibilities of participation in such programs can only be made by a doctor based on an individual assessment of the patient’s condition.

Treatment of such diseases requires a step-by-step approach:

1. confirmation of the diagnosis and genetic variant
2. assessment of the disease severity and the patient’s current state
3. determination of whether standard therapy, transplantation, or gene-cell treatment is suitable for the patient
4. organization of treatment in a specialized center
5. long-term observation after therapy

This path is especially important in rare blood diseases, where the result depends not only on the drug itself but also on the quality of the entire patient management program.