The story of the child Youssef Medhat, who has “Duchenne” muscular dystrophy and urgently needs to obtain a gene injection worth about EGP 150 million ($3 million), preoccupied public opinion during last August, so much so that the donation campaign opened in his name raised more than EGP 173 million in just 15 days, after it topped social media and was supported by celebrities, which earned it great humanitarian sympathy that accomplished the task of raising a huge sum in a matter of days.
After the Ministry of Social Solidarity, which is responsible for managing the campaign’s accounts, announced the closure of the accounts after succeeding in raising a sum exceeding the required amount by more than EGP 20 million, many questions emerged, some of them related to enquiries about: Did the child receive the treatment? And where did the money in excess of what was required go? And others were sceptical, such as: Is the child really ill and in need of the injection referred to? And will the treatment be of benefit in this case, and does it hold the necessary licences?
Among the questions raised by the campaign was also the point of raising a huge sum equivalent to $3 million to treat a single child, when this sum could treat thousands of children with other diseases, and another question about whether a patient must become a “trend” in order to obtain their right to health, and what about those who are unable to market their cases?
These questions, casting doubt on the fate of the donated money and questioning the usefulness of the treatment, stirred concern among citizens about their donations and about how deserving the cases they chose to donate to were. At the same time, they played with the hopes of many parents of children with muscular dystrophy who have followed the path of the child Youssef and are racing against time to raise the cost of the most expensive drug in the world through donations, especially as this approach had been rejected, before the child Youssef, by decision-makers in the state for several considerations.
Youssef’s Story Leads to a Protest in Front of the Presidency
Six years ago, with the first symptoms appearing on the child Mohamed, the son of Ahmed Awad, who lost his mother when he was no more than a year and a half old, the father began searching for the reasons behind the decline in the motor development of his son, who had started his life normally. When he reached the age of three, symptoms of weakness in movement began to appear on him, until it became clear to the father, through repeated tests, that he had Duchenne muscular dystrophy.
As Awad recounts to Zawia3, at that time there was no definitive treatment for the condition, only some measures that limit the progression of the disease, such as physiotherapy, hydrotherapy and doses of cortisone. Later the gene drug “Elevidys”, given as a single dose in a lifetime, appeared, but he was shocked when he learnt that its cost reaches about EGP 150 million ($3 million).
From here began the father’s journey to provide the treatment for his son, especially after he learnt that a condition for obtaining it is that the child is still able to move, and his son, who had reached six years of age, was still able to. He began submitting appeals for help to everyone holding responsibility in the state, starting with rights bodies such as the National Council for Human Rights and the National Council for Childhood and Motherhood, as well as the Ministry of Health and the Prime Minister’s Office, but to no avail. So he decided to take the legal route and filed a case against the Ministry of Health and the head of the General Health Insurance Organisation, and obtained a court ruling affirming the child’s entitlement to be provided with the drug.
After the court ruling, the father faced another problem: the refusal of the Supreme Committee for Muscular Dystrophy at the Ministry of Health to implement it, on the grounds that the treatment is unsuitable for the child’s condition and causes death. The father turned to the courts once again over the ministry’s refusal to implement the ruling, and obtained a second ruling. Now Mohamed is nine years old, and signs of motor delay have begun to appear on him that prevent him from obtaining the gene drug.
After all this, the father was surprised by the opening of an account to raise donations for the child Youssef, and asked in astonishment: “If the reason for refusing to provide the treatment is that it is unsuitable and causes death, how was approval given for the child to open an account?” The father and other parents began searching for the reason why a window of hope had been opened for one child while their own children are dying slowly, until they learnt that allowing donations of large sums to be raised requires a sovereign decision: “At Health Insurance they told us that their regulations do not allow the provision of treatment whose price exceeds EGP 1 million, after which the matter is referred to the Ministry of Health, whose powers stop at the limit of ten million, after which the matter is referred to the Prime Minister’s Office and then to the Presidency.”
This information was the reason that pushed a group of about 20 families of muscular dystrophy patients to take their requests to Al-Ittihadiya Presidential Palace, according to Ahmed Awad: “There, we could not even reach the palace gate, as the security forces intercepted us, took the papers from us and promised to deliver them. But the matter was hard on some of the mothers, who had been waiting for one of the officials to sit with them and hear about their daily suffering with their children, so they stood in front of the palace and began to cry, until some of those gathered were detained as a kind of threat. Then they let us go again, affirming that the requests would be delivered and examined. But in general there was sympathy with us, especially with the presence of our children, on whom signs of suffering are visible.”
The State Manages the Accounts
Mahmoud Fouad, human rights lawyer and director of the Egyptian Centre for the Right to Medicine, affirms to Zawia3 that in Egypt there is no room for doubting fundraising operations, as moving money has become a difficult and complicated procedure that falls entirely under the supervision of the state, pointing out that the individuals for whom donations are raised do not get their hands on the money at all, from the opening of the account to the donation campaign’s success in raising the sum and obtaining the drug.
Fouad explains that the process of opening an account to raise donations for the treatment of a disease goes through several stages: the patient’s family go to the Ministry of Social Solidarity to request the opening of an account, and the ministry in turn refers it to the Ministry of Health and then to a supreme committee specialising in the disease, which diagnoses the case precisely and determines its eligibility to obtain the treatment. The Ministry of Health then returns to the Ministry of Social Solidarity confirming the child’s eligibility for the treatment and its availability, after which Social Solidarity sends the case to Public Security, which makes enquiries about the child and his family and makes sure that no terrorist organisations stand behind the request and that there are no other cases, and returns to the Ministry of Social Solidarity with its approval, so that the latter begins the procedures for opening the account, whether at a national bank or through other collection mechanisms such as the “Cash” or “Fawry” services, and gives the patient’s family the account numbers.
He continues that the patient’s family then take charge of the advertising campaigns for donations, whether through social media, leaflets or any method that occurs to them. If the sum is completed, the bank closes the account and sends a notification to the Ministry of Social Solidarity, which writes to the Ministry of Health to transfer the money and import the treatment; the citizen is then notified that the drug has been imported and that he must go to the designated hospital to obtain it. Fouad comments: “All these procedures take place completely away from the citizen, who can do nothing but wait until the donation sum is complete and then go to the hospital to obtain the treatment only, and not a single piastre of the money reaches his hand.”
This was confirmed by Dr Ayman Abdel Mawgoud, Deputy Minister of Social Solidarity, in a televised statement, pointing out that Law No. 149 of 2019 regulating civil work includes an article regulating fundraising for a civil or charitable purpose for legal persons or individuals, and that the case of the child Youssef, which raised the donation sum, was subject to all the procedures regulating the opening of an account to raise donations and obtain the drug under the ministry’s supervision.
Sherifa Motawea, head of the Egyptian Association for Muscular Dystrophy Patients, confirms to Zawia3 what the director of the Egyptian Centre for the Right to Medicine said, pointing out that fundraising does not happen “with the stroke of a pen”, and that no citizen can open an account to raise donations on their own initiative. The crisis, however, is that these procedures, despite being in the patient’s interest, take an extremely long time, which may lead to the chance of treatment being lost if the condition deteriorates.
She adds that there are several problems facing parents in relation to muscular dystrophy, the first being the absence of a hospital in Egypt specialised in muscular dystrophy, and consequently there are not enough doctors specialised in the disease, especially if any case experiences emergency symptoms. The absence of a hospital also makes fundraising operations arduous and full of many successive steps, unlike if there were a single hospital that raised donations, determined the eligible patients and provided them with the drug.
The second problem lies in the mechanism by which the state deals with donations in the event that the account is closed before the specified sum is completed, or that the sum exceeds the required limit, as in the case of the child Youssef, or that the sum is successfully raised but the condition of the patient for whom it was raised deteriorates so that it is no longer possible for them to receive the treatment. Sherifa explains that in all these cases the money does not go to muscular dystrophy patients, but is directed to the Fund for Supporting the Projects of Civil Associations and Foundations of the Ministry of Social Solidarity, according to the law. She comments: “According to the law, the fundraising account is opened in the patient’s favour for one year, with the possibility of extension for a second year, and, for example, if EGP 149 million is raised out of the EGP 150 million, the child does not obtain the treatment, and the money is directed to the aforementioned fund.”
According to Article 65 of the executive regulations of the Law Regulating the Practice of Civil Work No. 149 of 2019, “the money remaining after the purpose of the collection has been achieved, or after it has become impossible to achieve, shall devolve to the Fund for Supporting the Projects of Civil Associations and Foundations, unless the permit issued in this regard specifies the body to which this money shall devolve.”
According to the chair of the board of the Egyptian Association for Muscular Dystrophy Patients, the third problem lies in the patient’s need to become a “trend” on social media in order to be able to raise money, which completely contradicts the right to health, as there are many cases that need treatment but do not know a way to spread among people: “There are currently more than 30 children with accounts opened for them, and their families are fighting to reach the trend.”
She points out that the child Youssef Medhat is the first child to succeed in raising the sum for purchasing the gene injection, whose price reaches $3 million, but there are other children seeking to raise money to no avail, and children raising donations to obtain a drug that stabilises the condition, whose price is lower than the gene injection, but which is taken annually, meaning a donation campaign every year, which is extremely arduous. According to the child Youssef’s lawyer, the child travelled to the State of Qatar to receive the gene therapy.
Sherifa Motawea continues that there is a second case of a child for whom an association in Menoufia opened an account to raise donations, but the sum did not exceed EGP 42 million, and not a single pound of it was spent, as he obtained full support from the State of Qatar and travelled to receive treatment there with this support: “The cases are known and we follow them, and even the cases raising donations to buy the stabilising drug we know about, and they open an account every year.”
Ahmed Awad, father of the child Mohamed, continues that accounts have now been opened for many patients, but who can raise EGP 150 million? And even the donors, how do they choose whom to donate to? And will the sick children hold out against time?: “Delaying the procedures means a sentence of slow death for our children; every day we wake up, we examine them to see which of their abilities they have lost,” pointing out that he is not convinced by the idea of creating donation accounts because they take time and do not reach the required sum without strong advertising campaigns that many people do not have, and if the accounts are closed before the sum is completed, it is transferred to the Ministry of Social Solidarity: “As if we are collecting for them.”
EGP 150 Million for One Child, or Do We Distribute It Among Children?
Dr Alaa Ghannam, a treatment policy expert and head of the Right to Health Unit at the Egyptian Initiative for Personal Rights, affirms to Zawia3 that all patients have an equal right to treatment, and that the high cost of treatment is no justification for leaving them to suffer. It would be better not to leave patients striving by themselves to raise the cost of their treatment, but for the state to redraw the rules for providing treatment through the rare diseases fund that has already been established, so that it serves as an umbrella covering all patients, instead of attempts at survival being individual.
In 2024, Law No. 5 was issued on adding rare and genetic diseases to the Medical Emergencies Fund established under Law No. 139 of 2021, which aims to contribute to the costs of disasters and medical emergencies, the treatment of genetic and rare diseases, and crises and epidemics.
Sherifa Motawea likewise affirms that medicine is a right and the recipient is a patient, and that the Egyptian Constitution guarantees everyone the right to treatment and care; therefore the matter of treatment should not be approached with the logic of “what takes priority? Spending a large sum on a single patient, or dividing it among a larger number of patients with other diseases”. In the end, it is not the fault of a muscular dystrophy patient that he contracted a rare disease whose treatment is costly.
Article 18 of the Egyptian Constitution stipulates: “Every citizen has the right to health and to comprehensive health care in accordance with quality standards. The state guarantees the maintenance and support of public health facilities that provide health services to the people, and works on raising their efficiency and their fair geographical distribution. The state commits to allocating a percentage of government spending to health of no less than 3% of the gross national product, which shall gradually increase until it is in line with global rates. The state commits to establishing a comprehensive health insurance system for all Egyptians covering all diseases, and the law regulates citizens’ contribution to its subscriptions or their exemption from them according to their income rates. Refusal to provide treatment in its various forms to any human being in cases of emergency or danger to life is criminalised.”
Regarding the efficacy of the drugs available for muscular dystrophy, Sherifa says that the treatment of muscular dystrophy, like any treatment, varies in patients’ response to it according to each case, but it represents hope that “Duchenne” will become a treatable disease and not a hopeless idea.
Muscular dystrophy of the “Duchenne” type is a rare genetic disease characterised by a mutation in the “dystrophin” gene, which causes gradual weakness and damage to the body’s muscles and ultimately leads to death. “Duchenne” affects one child in every 3,500 to 5,000 male births around the world, noting that cases of “Duchenne” with no genetic history have appeared, and cases among females have also appeared. Ahmed Awad says: “In our group that we communicate with, we have three girls who have been diagnosed with muscular dystrophy, but the scientific research on which the medical committees rely still limits the disease to males only.”
Sherifa Motawea explains that muscular dystrophy has more than 60 types, but the “Duchenne” and “spinal” types are the ones for which therapeutic drugs have begun to appear.
On the other hand, the researcher on the health file at the Egyptian Initiative for Personal Rights, Dr Ayman Sabae, says that decisions related to health are extremely difficult and cannot be governed by emotions alone, explaining that with regard to muscular dystrophy there are three questions related to the matter. The first is: Is the high-cost treatment effective, approved and suitable for all “Duchenne” cases? He explains that if the treatment is effective, then obtaining it is a right to health, but if it is not, which in his estimation applies to the high-cost gene injection, the state hesitates to guarantee the treatment and thinks with the logic of “Do I treat one child with EGP 150 million, or do I treat 150,000 children with common diseases with the same sum?”, commenting: “Thinking with this logic applies only in the case where the efficacy of the drug is not established.”
Sabae adds, speaking to Zawia3, that the gene injection is not approved as a treatment protocol in many countries, including Egypt, and that the US Food and Drug Administration (FDA) suspended its distribution at one point, continuing that there are “Duchenne” cases for which this gene therapy is not suitable, but it is difficult for parents to accept this idea, so they seek to obtain the treatment by any means, yet the matter is not a matter of hope only. This article documents later the precise regulatory status of the drug, as it obtained FDA approval with strict restrictions and warnings, not a complete withdrawal.
Muscular dystrophy patients are racing to raise the cost of a drug called Elevidys, the first gene therapy designed specifically to treat “Duchenne” muscular dystrophy. It consists of a single dose given to the patient by intravenous injection, to enable the body to produce a shortened form of the “dystrophin” protein (Micro-dystrophin) that Duchenne patients lack, thereby helping to protect the muscles from deterioration and atrophy and slowing the progression of the disease.
The US Food and Drug Administration (FDA) gave this drug preliminary approval in June 2023, on condition that it be given to children able to walk in the age group of four to five years, and in June 2024 the FDA expanded the approval to include all Duchenne patients from the age of four, on condition that there are no specific genetic mutations that conflict with the drug.
But in 2025 the drug faced a temporary suspension of distribution, as a result of deaths caused by acute liver failure being recorded in some patients. After some investigations and trials were carried out, provision of the drug was resumed for patients able to walk only, while its licence was withdrawn for patients unable to walk. The US Food and Drug Administration also obliged the manufacturer to add a “black box warning”, the most severe drug warning, to the drug’s packaging, warning of the risks of fatal acute liver failure, along with renewed protocols and strict periodic tests of liver function before and after receiving the treatment.
The FDA also recommended that health care providers instruct patients on the need to stay close to an appropriate health care facility, as determined by the health care provider, for a period of no less than two months after receiving the drug, which indicates that the cost of obtaining this drug is not limited to its price.
Although the drug is available in America to patients able to walk under medical supervision and strict safety conditions, and is also available in a limited number of countries, including some Gulf states such as the UAE, Qatar, Kuwait and Bahrain, it has not yet obtained approval from the Egyptian Drug Authority.
The high price of the drug, exceeding $3 million, represents a fundamental challenge to its spread and availability to patients, in addition to the continuation of clinical evaluation procedures.
Besides the gene therapy, there is another treatment that patients describe as “stabilising”, as it limits the deterioration of the condition: the drug “Duvyzat”, approved in the United States since 2024, and scientifically proven to treat specific cases of “Duchenne” muscular dystrophy in patients aged six years and above who are able to walk.
Although it is not a definitive treatment, it works as an enzyme inhibitor, which helps reduce inflammation, fibrosis and the accumulation of fat in the muscles, and stimulates the regeneration and repair of muscle fibres, which contributes to slowing the deterioration of the patient’s muscle strength. This drug represents a chronic treatment for the disease, with an average annual cost of about $700,000, which makes it more expensive than the gene drug that is given only once, while patients who need the stabilising drug are forced to open donation accounts annually in an attempt to raise the sum, equivalent to about EGP 35 million, which is extremely difficult.
Ahmed Awad points out that he filed a third lawsuit to obtain the stabilising treatment, after his son began to lose the ability to move and the gene therapy was no longer suitable for him, and obtained a ruling that has not yet been implemented; now the option before him is raising donations. He adds: “Opening an account to raise donations for this drug is a task whose hardship is greater than raising the sum for the definitive gene therapy, because this account must be renewed annually.”
Ahmed Awad is weighing whether to follow the path of those who opened donation accounts and await his fate, or to continue with the treatments available for his son Mohamed, such as physiotherapy and hydrotherapy: “Even this treatment is costly. I live in Fayoum Governorate, and to travel to Cairo I need a private car, then a series of scans, tests and treatments, and the cost each time may range between EGP 5,000 and EGP 10,000, which is difficult for many of us. Others have two or three sons suffering from the same disease, and as a form of solidarity we may help one another if one of us is unable to pay the cost of treating his children.”
As for the second question raised by Dr Ayman Sabae, the health researcher, it is: In the case of a patient who meets the conditions for obtaining the expensive treatment, does this treatment have effective and available alternatives? Or is it the only treatment available? He explains that some muscular dystrophy cases have treatments guaranteed by the state at a much lower cost, but they are periodic treatments and not given only once, like the gene therapy.
The health researcher continues that the third question that imposes itself is: Why does the state not guarantee the high-cost treatment if its efficacy is proven? Is it because of its high cost? Is there a capacity to contract at reasonable prices or to manufacture the drug locally? And what is the solution if this capacity is not available? He answers that in this case comes the role of the donation fund, which should not operate individually at all.
He explains that individual donation funds stand as a major obstacle to achieving justice in the distribution of money and the treatment of patients, as the matter depends on who has the ability to launch campaigns, manage marketing and appeal to people’s emotions, and the right to treatment should not be linked to whoever was able to elicit more sympathy; all this represents a violation of the right to health and a mismanagement of the resources of the state and of citizens.
Why Is There No Hospital and No Donation Fund?
All the sources who spoke to Zawia3 agreed on the importance of establishing a hospital for muscular dystrophy patients that specialises in and goes deep into the details of the disease and its different cases, and at the same time raises donations for patients and distributes them fairly in a way that guarantees everyone equal opportunities for treatment, so that if a certain sum is raised for a case that deteriorated before receiving treatment, the money goes to another case, not to the Ministry of Social Solidarity.
Sherifa Motawea explains that there are many ideas to support muscular dystrophy patients, such as allocating a plot of land to build a hospital that civil associations would undertake to establish, along the lines of Hospital 57357, Dr Magdi Yacoub’s hospital, the burns hospital and others, to provide treatment for all children and adults, conduct research, and launch advertising to raise donations and distribute them fairly among cases, in addition to activating the rare diseases fund with the state taking charge of promoting it: “The fund is not like individual accounts, because it will address businessmen, companies and bodies, and it may add monthly deductions from employees or small fees on bills, making it possible to treat all patients.”
Motawea points out that the number of muscular dystrophy cases registered with the Ministry of Health is about 2,000, and although the actual number across the country may be much higher, the Ministry of Health now knows of 2,000 cases, and trying to help them receive treatment is not a difficult task.
Mahmoud Fouad likewise affirms that establishing a hospital to treat the disease guarantees the continuity of treatment, pointing out that muscular dystrophy is an extremely difficult disease for all parties, starting with the pain the children suffer, through the suffering of their parents, and up to the high cost of its rare treatment. He points out that the centre has communicated extensively with the Ministry of Health and various bodies, and was able to move only the files of tests and scans, as a muscular dystrophy patient needs special and costly tests and scans every six months so that the disease does not progress suddenly, and some departments in university hospitals have indeed been opened to carry out the tests and scans free of charge.
In 2021, President Abdel Fattah El-Sisi launched an initiative to treat patients with “spinal” muscular atrophy, with the aim of providing diagnostic services and providing gene therapy and medical support to children free of charge at the state’s expense. The initiative undertook to provide screening, treatment and health awareness services and to provide genetic tests according to the case, but the dispensing of treatment was limited to children under six months.
The initiative provided sick children with the drug “Zolgensma”, the only one in the world given as a single dose for spinal muscular atrophy in children under two years of age, which works by increasing the amount of SMN protein in the body to maintain muscle functions properly, and the cost of a single dose ranges between $2 million and $3 million.
The director of the Egyptian Centre for the Right to Medicine says that the presidential initiative provided treatment for only 15 cases and then stopped due to the high costs, in addition to the crisis of providing dollars.
During their meeting with officials from the Ministry of Health, parents submitted proposals to provide the treatment by drawing on investors who would provide it inside Egypt in order to overcome the dollar crisis, and others to limit the disease by making genetic testing mandatory as part of the premarital examination, and carrying out ICSI procedures for those already married who have a genetic history of muscular dystrophy. Ahmed Awad says: “Genetic testing costs up to EGP 16,000, but if people knew the suffering that could befall them if they had a child with muscular dystrophy, and the millions they would need to treat the child, they would not hesitate to pay this cost. And even those already married who have a genetic history in the family, the state can make them aware of the importance of having children through ICSI procedures at one of the specialised government hospitals, such as Al-Hussein University Hospital, while bearing part of the cost of the procedure. All this would save the state and people a great deal, if there is a real desire to confront the problem.”
Dr Ayman Sabae calls for transparency and clarity with families regarding drugs and their suitability for each case, for the state to guarantee a drug where it is effective, and for a fund to be established that is responsible for raising donations and distributing them in an organised manner and according to disciplined protocols.
In sum, while acknowledging the difficulty of muscular dystrophy, the absence of a proven definitive treatment for it, and the high cost of the available treatment, which is still subject to restrictions and clinical evaluation, there are many duties that the government must perform towards citizens, starting with raising awareness of the disease and its seriousness, through opening horizons for discussion to find solutions that ease patients’ suffering, such as establishing a hospital based on donations and supported by the state, and activating the rare diseases fund and having it take charge of raising donations so as to give all patients an equal right to the money, among other solutions, instead of leaving patients searching for their chances of survival, each according to their own abilities.
