{"id":16924,"date":"2026-09-18T17:11:52","date_gmt":"2026-09-18T15:11:52","guid":{"rendered":"https:\/\/zawia3.com\/?p=16924"},"modified":"2026-10-07T04:53:21","modified_gmt":"2026-10-07T02:53:21","slug":"duchenne-muscular-dystrophy-egypt","status":"publish","type":"post","link":"https:\/\/zawia3.com\/en\/duchenne-muscular-dystrophy-egypt\/","title":{"rendered":"Duchenne Treatment in Egypt: Must a Child Go Viral to Receive Care?"},"content":{"rendered":"

Youssef Medhat\u2019s story dominated public attention in August. The child has Duchenne muscular dystrophy and was seeking urgent access to gene therapy priced at around EGP 150 million, or $3 million. A celebrity-supported donation campaign raised more than EGP 173 million in fifteen days.<\/p>\n

After the Social Solidarity Ministry, which supervised the accounts, announced closure once the target was exceeded by more than EGP 20 million, questions followed: had Youssef received treatment, where would the surplus go, was his need verified, and was the treatment effective and licensed for his condition?<\/p>\n

Other questions concerned equity: should $3 million treat one child rather than many with other illnesses? Must a patient become a social-media trend to secure healthcare, and what happens to families unable to market their cases?<\/p>\n

Those questions unsettled donors while affecting hopes of other families racing to raise the price of exceptionally expensive medicines. Before Youssef\u2019s campaign, this fundraising route had faced resistance from decision-makers for several reasons, families say.<\/p>\n

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Youssef\u2019s campaign and families at the presidential palace<\/h2>\n

Six years ago, Ahmed Awad began investigating the slowing motor development of his son Mohamed, whose mother died when he was eighteen months old. Mohamed initially developed normally, then showed weakness around age three. Repeated examinations identified Duchenne muscular dystrophy.<\/p>\n

Awad says there was then no definitive treatment, only measures such as physiotherapy, hydrotherapy and corticosteroids to limit progression. Elevidys later offered the possibility of a one-time gene therapy, but its roughly EGP 150 million price was a shock.<\/p>\n

Learning that walking ability affected eligibility, he sought help when his six-year-old could still walk. Appeals to the National Council for Human Rights, National Council for Childhood and Motherhood, Health Ministry and Cabinet brought no result, he says. He sued the ministry and Health Insurance Authority and obtained a judgment recognising his son\u2019s entitlement to the medicine.<\/p>\n

Awad says the ministry\u2019s supreme muscular-dystrophy committee refused implementation, considering the treatment unsuitable and citing death risks. He sued over non-implementation and obtained a second judgment. Mohamed is now nine and losing mobility, threatening his eligibility for gene therapy. Those accounts of judgments and committee decisions are the father\u2019s testimony.<\/p>\n

Youssef\u2019s approved fundraising account made him ask: if officials refused treatment as unsuitable and dangerous for his son, why approve a campaign for another child? Families were told that insurance regulations stop at EGP 1 million, ministry authority at EGP 10 million, and larger sums require escalation to the Cabinet and presidency. These thresholds are what Awad says families were told.<\/p>\n

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About twenty families took their requests to the presidential palace at Ittihadiya. Awad says security officers collected their documents and promised delivery, while mothers hoping to speak directly to officials wept outside. Some people were briefly held as a warning, he says, then released with assurances their requests would be reviewed. He also describes sympathy, particularly because their children\u2019s suffering was visible.<\/p>\n<\/blockquote>\n

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The state controls fundraising accounts<\/h2>\n

Mahmoud Fouad, rights lawyer and director of the Egyptian Centre for the Right to Medicine, says donation-account operations are complex and wholly state-supervised. Families do not control the money between opening an account and purchasing treatment, he argues.<\/p>\n

He describes an application to the Social Solidarity Ministry, referral to the Health Ministry and a specialist committee to diagnose the patient and assess treatment eligibility and availability, followed by security checks on the child and family. After approval, accounts can be opened through a national bank or payment channels such as Cash or Fawry, and details given to the family.<\/p>\n

Families then publicise appeals through social media, printed material or other means. Once the target is reached, the bank closes the account and informs the ministry, which contacts the Health Ministry to transfer funds and import the medicine. The patient is notified to attend the designated hospital.<\/p>\n

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\u201cThe citizen can only wait for the donation target and go to hospital for treatment; not one piastre of the money reaches their hands,\u201d Fouad says.<\/p>\n<\/blockquote>\n

Social Solidarity Ministry official Ayman Abdel Mawgoud likewise said in televised remarks that Civil Work Law No. 149 of 2019 regulates charitable fundraising by organisations and individuals and that Youssef\u2019s case passed the required procedures under ministry supervision.<\/p>\n

Sharifa Motawa, chair of the Egyptian Muscular Dystrophy Patients Association, says no citizen can simply open a fundraising account on their own. While oversight protects patients, the procedures can take so long that deterioration closes a treatment window.<\/p>\n

She identifies the lack of a specialised muscular-dystrophy hospital and sufficient specialists as one problem, especially during emergencies. A single institution could coordinate donations, eligibility and medicines instead of leaving families to navigate many sequential steps.<\/p>\n

A second problem is what happens when an account closes short of its target, raises a surplus or reaches its target after the intended patient deteriorates beyond eligibility. Motawa says the money generally goes to the ministry\u2019s Fund to Support Association and Civil Foundation Projects rather than directly to another muscular-dystrophy patient.<\/p>\n

She says accounts are permitted for a year, extendable for another, and gives the example of EGP 149 million collected against a EGP 150 million target: the child would still not receive treatment and funds would be redirected. The relevant permit and law govern the actual destination.<\/p>\n

Article 65 of the executive regulations states that funds remaining after the collection purpose is achieved or becomes impossible pass to the association-projects fund unless the authorisation specifies another recipient. That exception matters: redirection is not necessarily identical for every permit.<\/p>\n

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A third problem is dependence on going viral. \u201cMore than thirty children currently have accounts, and their families are fighting to become a trend,\u201d Motawa says. Many patients need care without knowing how to reach a large public audience.<\/p>\n<\/blockquote>\n

She describes Youssef as the first child to successfully raise the roughly $3 million gene-therapy target through this route. Others seek donations without success, including for continuing treatment requiring renewed fundraising every year. According to Youssef\u2019s lawyer, he travelled to Qatar for gene therapy.<\/p>\n

Motawa also describes another child whose account, opened through a Menoufia association, raised EGP 42 million but spent none because Qatar fully supported treatment there. She says the association follows these cases, including annually renewed campaigns for ongoing medicine.<\/p>\n

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Awad questions who can raise EGP 150 million, how donors choose among children and whether patients can withstand the wait. \u201cEvery morning we look to see which abilities our children have lost,\u201d he says. He distrusts a route requiring publicity resources many families lack, particularly if incomplete accounts ultimately redirect funds elsewhere: \u201cIt is as though we are collecting for them.\u201d<\/p>\n<\/blockquote>\n

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One expensive treatment, or care for many?<\/h2>\n

Alaa Ghannam, health-policy expert and head of the Egyptian Initiative for Personal Rights\u2019 right-to-health programme, says all patients equally deserve treatment and high cost is no justification for abandoning them. Rather than individual rescue campaigns, he calls for the state to use the rare-disease fund as an umbrella for everyone.<\/p>\n

Law No. 5 of 2024 added rare and hereditary diseases to the Medical Emergency Response Fund established by Law No. 139 of 2021, supporting disaster and emergency care, hereditary and rare diseases, crises and epidemics.<\/p>\n

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Motawa rejects framing the issue simply as one costly patient versus many cheaper ones. A patient is not to blame for having a rare disease with an expensive treatment, she says, and constitutional healthcare rights apply to them too.<\/p>\n<\/blockquote>\n

Article 18 protects health and comprehensive quality healthcare, preservation and equitable geographical expansion of public health facilities, government health spending of at least 3% of gross national product with gradual increases, and universal insurance covering all diseases with contributions or exemptions based on income. It also criminalises refusal of treatment in emergencies or life-threatening circumstances.<\/p>\n

Motawa says treatment response varies individually, as with other medicines, but new therapies offer hope that Duchenne can be treated rather than regarded as hopeless.<\/p>\n

Duchenne is a rare genetic disorder involving the dystrophin gene, causing progressive muscle weakness and damage and potentially early death. The report cites approximately one affected child per 3,500\u20135,000 male births. Cases can occur without known family history, and females can rarely be affected.<\/p>\n

Awad says their support group includes three girls diagnosed with muscular dystrophy and criticises committees that focus only on males. Those diagnoses are described as muscular dystrophy generally, and do not establish that each girl has Duchenne. Motawa says there are more than sixty forms of muscular dystrophy and highlights new therapies for Duchenne and spinal muscular atrophy; the latter is a distinct neuromuscular condition, not a subtype of Duchenne.<\/p>\n

EIPR health researcher Ayman Saba argues that difficult healthcare decisions cannot be governed by emotion alone. His first question is whether a high-cost therapy is effective, approved and suitable for a particular patient. Where effectiveness is established, access is a right, he says; uncertainty complicates decisions about spending EGP 150 million on one child or treating large numbers with common conditions.<\/p>\n

Saba questions the evidence for the expensive gene therapy and notes that it is not included in treatment protocols everywhere, including Egypt. He points to a temporary US distribution interruption and says it is unsuitable for some Duchenne patients, a possibility difficult for parents to accept. His reservations should be distinguished from the FDA\u2019s documented approval with restrictions, rather than a complete withdrawal.<\/p>\n

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Therapies, eligibility and regulatory warnings<\/h2>\n

Elevidys is a single intravenous gene therapy designed to enable production of a shortened dystrophin protein, micro-dystrophin. It is intended to address the protein deficiency underlying Duchenne; it should not be described as a proven complete cure or a guaranteed outcome.<\/p>\n

It received accelerated FDA approval in June 2023 for ambulatory children aged four to five, then expanded approval in June 2024 for patients aged four and older. Eligibility also depends on confirmed genetic diagnosis and specific contraindications, not age alone.<\/p>\n

In 2025, distribution was temporarily interrupted amid serious safety concerns. On 28 July 2025, the FDA recommended ending the voluntary hold for ambulatory patients, while a hold remained for non-ambulatory patients following two deaths. FDA notice<\/a>.<\/p>\n

On 14 November 2025, the FDA restricted the indication to ambulatory patients aged four or older with a confirmed DMD-gene mutation and removed the non-ambulatory indication. It added a boxed warning for serious liver injury and acute liver failure, including fatal outcomes, alongside stricter precautions and monitoring. Patients are to remain near an appropriate healthcare facility, as determined by their provider, for at least two months after infusion. FDA safety communication<\/a>.<\/p>\n

That follow-up means the medicine\u2019s price is not the entire cost of receiving care. The Arabic report says access exists in a limited number of countries, including some Gulf states, but that Egyptian Drug Authority approval had not been obtained at publication.<\/p>\n

A price exceeding $3 million and continuing clinical assessment remain major access barriers. The EGP 150 million campaign target is an approximate local cost cited by families, rather than a universal purchase price.<\/p>\n

Another treatment, described by families as a \u201cstabiliser,\u201d is Duvyzat, or givinostat. FDA-approved on 21 March 2024 for Duchenne patients aged six and older, it is a histone deacetylase inhibitor. The pivotal trial enrolled ambulatory boys on stable corticosteroids and showed less worsening in a muscle-function test. The approval\u2019s age-based indication should not be conflated with the trial\u2019s ambulatory population. FDA trial and approval summary<\/a>.<\/p>\n

The report describes effects on inflammation and muscle degeneration rather than a cure. It cites an estimated annual cost near $700,000, around EGP 35 million, requiring repeated fundraising. Those are reported estimates, not independently verified quotations; whether continuing treatment exceeds a one-time therapy\u2019s cost depends on duration.<\/p>\n

Awad says he obtained a third, still-unimplemented judgment seeking ongoing treatment after Mohamed began losing mobility and gene therapy ceased to be suitable. \u201cFundraising for this medicine is even more exhausting, because the account must be renewed annually,\u201d he says.<\/p>\n

He weighs fundraising against continuing physiotherapy and hydrotherapy, themselves expensive. From Fayoum he needs a private car to Cairo, then imaging, tests and care, costing EGP 5,000\u201310,000 per visit. Some families have two or three affected children and support one another when treatment payments become impossible.<\/p>\n

Saba\u2019s second question is whether a patient eligible for expensive therapy has effective, available alternatives. Some neuromuscular conditions have cheaper state-supported treatments, he says, although they may require repeated dosing rather than a one-time intervention.<\/p>\n

His third question is why the state would not fund a proven effective expensive treatment: can it negotiate reasonable prices or support local manufacture, and what should happen if those options are unavailable? A donation fund then has a role, he argues, but should not operate through isolated individual campaigns.<\/p>\n

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Individual fundraising undermines equitable allocation, Saba says, because success depends on publicity, marketing and eliciting sympathy. Healthcare entitlement should not depend on who can appeal most effectively to donors; he considers that unfair and a poor use of public and private resources.<\/p>\n<\/blockquote>\n

Sources interviewed by Zawia3 agree on the value of a specialised hospital with expertise across neuromuscular conditions, coordinated fundraising and fair allocation. If one intended recipient deteriorates before treatment, money could support another eligible patient rather than leaving the disease-care system.<\/p>\n

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A hospital and a collective fundraising system<\/h2>\n

Motawa proposes land for a charity-built, state-supported hospital on models such as 57357, Magdi Yacoub\u2019s hospital and burns hospitals, providing child and adult care, research and organised fundraising. She also wants activation and promotion of the rare-disease fund.<\/p>\n

Unlike individual accounts, such a fund could approach businesses, companies and institutions and consider voluntary or legally established contribution mechanisms, including payroll contributions or small bill levies, she suggests. These are proposals, not existing deductions.<\/p>\n

She says the Health Ministry has registered around 2,000 muscular-dystrophy cases, while the true number may be much larger. She considers organised assistance for the known patients feasible.<\/p>\n

Fouad says a dedicated hospital would sustain care for a condition painful for children and families and expensive to treat. His centre repeatedly contacted the ministry and other bodies but achieved movement mainly on tests and imaging, which patients need roughly every six months. Some university-hospital departments now provide those investigations free, he says.<\/p>\n

In 2021, President Abdel Fattah al-Sisi launched an initiative for spinal muscular atrophy, offering diagnosis, gene therapy, medical support and genetic tests free at state expense. The report says treatment under that initiative was limited to infants younger than six months.<\/p>\n

The initiative used Zolgensma, a one-time intravenous gene therapy for eligible young children with spinal muscular atrophy. The US indication is for children under two with bi-allelic SMN1 mutations; this is distinct from the reported Egyptian initiative\u2019s six-month threshold. The report estimates EGP-equivalent costs of $2\u20133 million a dose. FDA approval information<\/a>.<\/p>\n

Fouad says the presidential initiative treated only fifteen cases before stopping because of cost and foreign-currency shortages. That account is attributed to him rather than presented as an independently verified official termination announcement.<\/p>\n

In meetings with ministry officials, parents proposed local supply through investors to ease foreign-currency obstacles, genetic testing as part of premarital screening and assisted reproduction with genetic assessment for families with inherited risk. These proposals require individual genetic counselling and medical assessment, rather than assuming one reproductive approach fits every family.<\/p>\n

Awad says genetic testing can cost EGP 16,000 but believes awareness of the disease\u2019s burden would encourage screening. For already married families with inherited risk, he proposes awareness and partially subsidised treatment through specialist public hospitals such as Al-Hussein University Hospital. He argues prevention and support could reduce suffering and expense.<\/p>\n

Saba calls for honest, transparent explanations of medicines\u2019 suitability and effectiveness, state coverage where benefit is established, and a central donation fund allocating resources through disciplined protocols.<\/p>\n

The disease is difficult, available therapies are expensive and subject to eligibility limits, safety risks and continued assessment, and there is no established complete cure. Government responsibilities nevertheless include awareness, open discussion, specialised care and a functioning rare-disease fund that offers equal access to resources, rather than leaving every family to seek rescue according to its publicity skills.<\/p>\n","protected":false},"excerpt":{"rendered":"

A campaign raising EGP 173 million for one child exposes unequal access to rare-disease treatment, slow procedures and families\u2019 calls for collective support.<\/p>\n","protected":false},"author":4,"featured_media":16375,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"om_disable_all_campaigns":false,"footnotes":""},"categories":[413],"tags":[],"kateb":[15844],"class_list":["post-16924","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-society-en","kateb-yasmin-ali"],"jetpack_featured_media_url":"https:\/\/zawia3.com\/wp-content\/uploads\/2026\/09\/\u0636\u0645\u0648\u0631-\u0639\u0636\u0644\u0627\u062a.png","_links":{"self":[{"href":"https:\/\/zawia3.com\/en\/wp-json\/wp\/v2\/posts\/16924","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/zawia3.com\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/zawia3.com\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/zawia3.com\/en\/wp-json\/wp\/v2\/users\/4"}],"replies":[{"embeddable":true,"href":"https:\/\/zawia3.com\/en\/wp-json\/wp\/v2\/comments?post=16924"}],"version-history":[{"count":1,"href":"https:\/\/zawia3.com\/en\/wp-json\/wp\/v2\/posts\/16924\/revisions"}],"predecessor-version":[{"id":16925,"href":"https:\/\/zawia3.com\/en\/wp-json\/wp\/v2\/posts\/16924\/revisions\/16925"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/zawia3.com\/en\/wp-json\/wp\/v2\/media\/16375"}],"wp:attachment":[{"href":"https:\/\/zawia3.com\/en\/wp-json\/wp\/v2\/media?parent=16924"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/zawia3.com\/en\/wp-json\/wp\/v2\/categories?post=16924"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/zawia3.com\/en\/wp-json\/wp\/v2\/tags?post=16924"},{"taxonomy":"kateb","embeddable":true,"href":"https:\/\/zawia3.com\/en\/wp-json\/wp\/v2\/kateb?post=16924"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}