{"id":1497,"date":"2026-08-16T22:27:43","date_gmt":"2026-08-16T19:27:43","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=1497"},"modified":"2026-08-16T22:54:09","modified_gmt":"2026-08-16T19:54:09","slug":"frequently-asked-questions-about-elevidys-used-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/es\/frequently-asked-questions-about-elevidys-used-for-duchenne-muscular-dystrophy\/","title":{"rendered":"Preguntas frecuentes sobre el Elevidys utilizado para DMD"},"content":{"rendered":"\n<p class=\"wp-block-paragraph\">Duchenne muscular dystrophy (DMD) is a genetic condition that causes progressive muscle weakness. Boys are more likely to develop DMD, with symptoms often appearing in early childhood. DMD has no known cure. Medical professionals use physical therapy, steroids, and assistive technologies such as wheelchairs and braces to manage symptoms. New medications targeting the underlying genetic cause are being researched to help slow the progression of DMD. How effective is Elevidys at maintaining muscle strength in people with DMD? Let\u2019s take a closer look together. [<strong>Read more:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/what-is-duchenne\/\" data-type=\"post\" data-id=\"588\" target=\"_blank\" rel=\"noreferrer noopener\">What is Duchenne Muscular Dystrophy?<\/a>]<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Under the FDA\u2019s expedited approval procedure, which allows drugs to be approved for serious conditions when there is evidence suggesting a high likelihood of clinical benefit, Elevidys was authorized in 2023. Since then, the therapy has been closely monitored. Results from a confirmatory trial published by Sarepta Therapeutics in October indicated that, although the therapy did not meet its primary objective of improving children\u2019s mobility, it showed benefits on several secondary measures.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">In 2024, the FDA stated that Elevidys had received traditional approval for ambulatory individuals aged 4 years and older with a confirmed mutation in the DMD gene, and accelerated approval for non-ambulatory individuals aged 4 years and older with the same mutation. According to the EPA, there is insufficient safety data to justify its use in children younger than four. [<a href=\"https:\/\/dmdwarrior.com\/in-which-countries-is-elevidys-approved-is-elevidys-approved-in-europe\/\" target=\"_blank\" rel=\"noreferrer noopener\">In Which Countries Is Elevidys Approved? Is Elevidys Approved In Europe?<\/a>]<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>Things to Know About Elevidys<\/h2><nav><ul><li class=\"\"><a href=\"#cost-of-elevidys-gene-therapy\">Cost of Elevidys Gene Therapy<\/a><\/li><li class=\"\"><a href=\"#how-does-elevidys-work\">How Does Elevidys Work?<\/a><\/li><li class=\"\"><a href=\"#how-should-i-use-this-medicine\">How Should I Use This Medicine?<\/a><\/li><li class=\"\"><a href=\"#how-was-it-studied-for-dmd\">How Was It Studied for DMD?<\/a><\/li><li class=\"\"><a href=\"#which-exons-is-eligible-for-elevidys-which-exon-deletions-is-elevidys-not-suitable-for-who-should-not-use-elevidys\">Which Exon Deletions Are Eligible for Elevidys?<\/a><\/li><li class=\"\"><a href=\"#which-exon-deletions-is-elevidys-not-suitable-for\">Which Exon Deletions Are Not Suitable for Elevidys?<\/a><\/li><li class=\"\"><a href=\"#does-elevidys-work\">Does Elevidys work?<\/a><\/li><li class=\"\"><a href=\"#which-advantages-were-found-in-the-research\">What Benefits Were Found in the Research?<\/a><\/li><li class=\"\"><a href=\"#when-will-the-medicine-start-working\">When Will the Medicine Start Working?<\/a><\/li><li class=\"\"><a href=\"#are-there-reasons-this-medicine-should-not-be-used\">Who Is Not Eligible for Elevidys Gene Therapy?<\/a><\/li><li class=\"\"><a href=\"#what-monitoring-or-blood-tests-might-i-need\">What Monitoring or Blood Tests Might I Need?<\/a><\/li><li class=\"\"><a href=\"#what-types-of-interactions-can-happen\">What Types of Interactions Can Happen?<\/a><\/li><li class=\"\"><a href=\"#frequently-asked-questions-about-elevidys\">Frequently Asked Questions About Elevidys<\/a><ul><li class=\"\"><a href=\"#faq-question-1764153083449\">Could a child who has received Elevidys receive another (different) gene therapy in the future?<\/a><\/li><li class=\"\"><a href=\"#faq-question-1728832771702\">Is Sarepta aware that the cost of this gene therapy is not covered in many countries?<\/a><\/li><li class=\"\"><a href=\"#faq-question-1728833081790\">Why is the cost of Elevidys treatment not reduced?<\/a><\/li><li class=\"\"><a href=\"#faq-question-1728833604125\">Elevidys should review its pricing policy! Why is it so expensive?<\/a><\/li><li class=\"\"><a href=\"#faq-question-1728833862440\">Sarepta must decide between money and the children&#8217;s lives?<\/a><\/li><li class=\"\"><a href=\"#faq-question-1728832387311\">In which countries has Roche, which has the right to market the Elevidys gene therapy, applied for a licence?<\/a><\/li><li class=\"\"><a href=\"#faq-question-1739736490593\">Is the Roche Applying for Licenses for Gene Therapy in Every Country?<\/a><\/li><li class=\"\"><a href=\"#faq-question-1728832580723\">Why is antibody testing only available in the United States?<\/a><\/li><li class=\"\"><a href=\"#faq-question-1728833974425\">After receiving Elevidys gene therapy, if its effectiveness decreases or stops completely, can a second therapy be administered? Will the cost of this be covered free of charge?<\/a><\/li><li class=\"\"><a href=\"#faq-question-1761134853094\">Are Becker muscular dystrophy patients eligible for Elevidys gene therapy?<\/a><\/li><li class=\"\"><a href=\"#faq-question-1728831673732\">If the Elevidys gene therapy doesn&#8217;t work, will Sarepta reimburse patients for the cost of the drug?<\/a><\/li><li class=\"\"><a href=\"#faq-question-1741357550389\">Who Should Not Use Elevidys?<\/a><\/li><li class=\"\"><a href=\"#faq-question-1761047506785\">Why Was Elevidys Not Approved by the European Medicines Agency (EMA)?<\/a><\/li><\/ul><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 id=\"cost-of-elevidys-gene-therapy\" class=\"wp-block-heading\">Cost of Elevidys Gene Therapy<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">One of the most expensive medications in the world, ELEVIDYS costs around 2.91 million USD per patient when administered as a single intravenous infusion. The fact that the drug does not completely eliminate Duchenne muscular dystrophy, while carrying such a high price, places a significant financial burden on families and makes the treatment unaffordable for many.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Moreover, Sarepta has not yet applied for a license for the gene therapy in many countries.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Even if families with children who have Duchenne muscular dystrophy somehow manage to cover the cost, they may be forced to travel to countries such as the USA or the UAE. Families who need to stay abroad for three to four months for treatment may also face substantial additional travel and living expenses.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">It doesn\u2019t end there. Families may also need to travel to the USA or the UAE for one or two days to undergo an antibody test to determine whether ELEVIDYS is suitable for their child.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Except for some insurance coverage in the US, ELEVIDYS is not provided free of charge in other countries. Families do not want to wait while their children\u2019s condition progresses.<\/p>\n\n\n\n<h2 id=\"how-does-elevidys-work\" class=\"wp-block-heading\">How Does Elevidys Work?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Dystrophin is a protein that is essential for muscles to function properly and maintain their strength. DMD is caused by inherited mutations that prevent the body from producing functional dystrophin. A mutation is a change in the genetic code that the body uses to build its components. When dystrophin levels are too low, muscles gradually deteriorate. Weakness typically begins in the legs and pelvis, making it difficult to run, climb stairs, or get up from the floor. DMD can eventually affect the heart, lungs, and arms.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">DMD is a progressive condition that is typically diagnosed in early childhood. As children get older, the symptoms become more apparent. Since DMD has no known cure, medical professionals use medications to manage symptoms and physical therapy to help maintain muscle strength.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Some newer therapies have been developed to target specific genetic abnormalities or to deliver a dystrophin-related protein directly to muscle cells. This can enable muscle cells to produce a dystrophin protein or dystrophin-like protein. Higher levels of dystrophin may help muscles maintain their strength over time.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">One such treatment, commonly known as gene therapy, is ELEVIDYS. ELEVIDYS delivers genetic material to muscle cells using a modified virus known as a vector. It enables the body to produce a shortened form of the dystrophin protein, which may help slow disease progression.<\/p>\n\n\n\n<h2 id=\"how-should-i-use-this-medicine\" class=\"wp-block-heading\">How Should I Use This Medicine?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">ELEVIDYS is a one-time treatment. A healthcare professional administers it as an intravenous infusion, meaning the medication is delivered directly into a vein. To ensure close monitoring, the infusion is administered in a hospital. The infusion may take several hours. To prepare for the treatment, your healthcare professional may perform blood tests before the procedure.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">To help prevent infusion-related adverse effects, your healthcare provider will administer a corticosteroid medication. Before receiving ELEVIDYS, make sure all of your vaccinations are up to date.<\/p>\n\n\n\n<h2 id=\"how-was-it-studied-for-dmd\" class=\"wp-block-heading\">How Was It Studied for DMD?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">The effectiveness and safety of ELEVIDYS for the treatment of DMD have been examined in three studies. A placebo-controlled design, used in Studies 1 and 3, allows researchers to compare the effects of a therapy with those of a placebo to identify potential benefits and harms. Participants in the studies had specific DMD gene mutations. The studies shared several common characteristics.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">All participants (100%) were male, ranging in age from 4 to 8 years, with an average age of 6 years and an average weight of 22.6 kg.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The research found that 76% of participants who received ELEVIDYS were White, while 12% identified as Asian, 7% as other, 2% as African American or Black, 2% as \u201cnot reported,\u201d and 1% as belonging to mixed racial groups. Before receiving the infusion, every participant took a corticosteroid medication, and their antibody levels were below the threshold considered safe for the ELEVIDYS infusion.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">In the first study, participants received a single infusion of either a placebo or ELEVIDYS during the first phase. The placebo infusion contained no active medication. Participants were monitored for 48 weeks for changes in their condition and for adverse events. Following the first 48 weeks of the trial, participants were randomized to receive either placebo or ELEVIDYS, in the reverse order of what they had received during the first phase. Everyone in the study was ambulatory, meaning they could walk independently without assistance.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Every participant in Trial 2 received ELEVIDYS. There was no placebo group. Only 17% of the participants were non-ambulatory, meaning they were no longer able to walk independently. <strong>Learn More<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/biomarkers-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Why Creatine Kinase (CK), AST, ALT, and Dystrophin Levels Should Be Disclosed<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">In Study 3, participants received either an ELEVIDYS or placebo infusion.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The efficacy of ELEVIDYS in the studies was assessed by analyzing changes in the NSAA (North Star Ambulatory Assessment) score and\/or the amount of micro-dystrophin protein present in skeletal muscle. The NSAA is a test used to assess how well a person with DMD can move and perform daily activities. The highest possible score is 34. A lower score indicates greater limitations in mobility and physical function.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Across all trials, the average NSAA score was between 21 and 23, indicating some movement limitations while participants were still able to perform most everyday activities. Other measures of movement capacity included timed tasks such as sprinting or walking a certain distance, climbing four flights of stairs, and rising from a horizontal position.<\/p>\n\n\n\n<h2 id=\"which-exons-is-eligible-for-elevidys-which-exon-deletions-is-elevidys-not-suitable-for-who-should-not-use-elevidys\" class=\"wp-block-heading\">Which Exon Deletions Are Eligible for Elevidys?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">When reviewing ELEVIDYS Safety Information, you can see warnings concerning deletions in exons 1 to 17 and\/or exons 59 to 71. <strong>Patients with certain mutations involving these exons may be at risk of severe immune-mediated myositis.<\/strong><\/p>\n\n\n\n<h2 id=\"which-exon-deletions-is-elevidys-not-suitable-for\" class=\"wp-block-heading\">Which Exon Deletions Are Not Suitable for Elevidys?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Individuals with certain types of mutations, including any deletion in exon 8 and\/or exon 9 of the DMD gene, should not receive ELEVIDYS.<\/p>\n\n\n\n<h2 id=\"does-elevidys-work\" class=\"wp-block-heading\">Does Elevidys work?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">We are reaching out to families of children who have received ELEVIDYS gene therapy. We have spoken with some of them and asked them to share their experiences before and after treatment.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Families of children who have received ELEVIDYS gene therapy have reported noticeable differences in their children. They say that children who previously had difficulty climbing stairs can now overcome obstacles more quickly and without support.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">However, one of the biggest concerns for families is how long the effects of gene therapy will last.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">A review of ELEVIDYS published in The Washington Post raised concerns about the gene therapy.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cA follow-up randomized trial of 125 patients was designed to show that this change was meaningful and that the therapy could improve patients\u2019 scores on a 34-point scale called the North Star Ambulatory Assessment. [Source: Sarepta] But the drug did not demonstrate a clear benefit for patients in this randomized trial: While the gene therapy group\u2019s scores improved on average by 2.57 points, the placebo group\u2019s scores improved by 1.92 points, which was not statistically different.\u201d [<strong>Read more:<\/strong> <a href=\"https:\/\/www.washingtonpost.com\/opinions\/2024\/07\/22\/fda-gene-therapy-elevidys\/\" target=\"_blank\" rel=\"noopener\">Washington Post<\/a>]<\/p>\n\n\n\n<h2 id=\"which-advantages-were-found-in-the-research\" class=\"wp-block-heading\">What Benefits Were Found in the Research?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">In the studies, people who received ELEVIDYS experienced improvements in timed movement tests, increases in dystrophin levels in their muscles, and improvements in their NSAA scores. Compared with those who received a placebo, participants receiving ELEVIDYS showed some improvements in muscle function and mobility.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Changes in NSAA scores. At the end of the studies, the NSAA scores of participants who received ELEVIDYS improved by an average of two points, while those who received a placebo experienced a slight decline in scores. This suggests that ELEVIDYS may improve functional mobility in some patients. Younger children (ages 4\u20137) who received ELEVIDYS showed a greater change in their scores.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Changes in dystrophin levels. ELEVIDYS delivers genetic material that enables muscle cells to produce a shortened form of dystrophin. The amount of dystrophin in muscle cells increased in two studies. The reported levels were consistent with those found in healthy muscle. This suggests that higher levels of dystrophin may help protect muscles from further damage. More research is needed to determine how long this effect may last.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The study results showed that participants receiving ELEVIDYS improved their performance on timed activities compared with those receiving a placebo. Participants receiving ELEVIDYS were able to complete these activities slightly faster. More research is needed to determine how long these effects last.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">It is possible that your results may not match those observed in clinical trials. Whether the benefits outweigh the potential risks will need to be determined over time.<\/p>\n\n\n\n<h2 id=\"when-will-the-medicine-start-working\" class=\"wp-block-heading\">When Will the Medicine Start Working?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">It may take time to observe the effects of ELEVIDYS. Muscle function may change as the body begins to produce dystrophin in muscle cells. Your ability to walk, climb stairs, and get up from the floor may change over time. Monitoring your progress and discussing it with your healthcare professional are important. If you have concerns about your progress, contact your doctor.<\/p>\n\n\n\n<h2 id=\"are-there-reasons-this-medicine-should-not-be-used\" class=\"wp-block-heading\">Who Is Not Eligible for Elevidys Gene Therapy?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">If you have certain genetic mutations, such as a deletion of exon 8 or exon 9 in the DMD gene, you should not receive ELEVIDYS. Your healthcare professional may perform genetic testing to screen for these specific genetic alterations before prescribing ELEVIDYS.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Antibodies against the ELEVIDYS vector, AAVrh74, can affect eligibility for treatment. These antibodies may impair ELEVIDYS\u2019s ability to reach muscle cells. Before the infusion, your healthcare professional may perform a blood test to measure antibody levels. ELEVIDYS should not be used if the antibody level is above the specified threshold.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">If you have an active infection, such as a common cold, flu, stomach infection, ear infection, or lung infection, the corticosteroid treatment given before and after ELEVIDYS may increase the risk of serious side effects. If you have an ongoing infection, you should not begin the ELEVIDYS infusion or corticosteroid treatment until your healthcare professional advises that it is safe. If you have any of the following signs of infection, tell your doctor:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Fever or chills<\/li>\n\n\n\n<li>Body aches<\/li>\n\n\n\n<li>Coughing or trouble breathing<\/li>\n\n\n\n<li>Tiredness<\/li>\n\n\n\n<li>Sneezing<\/li>\n\n\n\n<li>Runny nose<\/li>\n\n\n\n<li>Sore throat<\/li>\n<\/ul>\n\n\n\n<h2 id=\"what-monitoring-or-blood-tests-might-i-need\" class=\"wp-block-heading\">What Monitoring or Blood Tests Might I Need?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">In order to monitor your health prior to the infusion and determine how your body reacts to the medication, your healthcare professional may order laboratory tests. Important components of that monitoring are the following.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Genetic testing.<\/strong> Before prescribing Elevidys, your doctor could do a blood test to check for the dystrophin gene. Additionally, they will check for certain mutations to determine whether Elevidys should be taken or not.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Antibodies to the vector of Elevidys.<\/strong> To determine the quantity of antibodies to the Elevidys vector (AAVrh74) in your blood, your doctor may do a blood test. This aids in determining the appropriate dosage and checks for any potential safety issues.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Infections.<\/strong> If you are infected, you should not start Elevidys. A blood test called a complete blood count (CBC) can be used to look for infections or other blood conditions.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Liver wellness.<\/strong> Your doctor might decide to postpone to start Elevidys until your liver issues are resolved if you have active liver disease or if you have yellowing of the skin or the whites of your eyes. Liver damage is detected via a liver function test (LFT). Your healthcare practitioner will review these tests following the infusion to monitor for any adverse effects on the liver.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Heart muscle wellness.<\/strong> Inflammation brought on by DMD has the potential to harm cardiac muscle cells. An intracellular protein known as troponin is released into your bloodstream by injured cardiac muscle cells. Before and after administering Elevidys, your doctor may measure your troponin levels to keep an eye on the condition of your heart muscle. If you have breathlessness or chest pain, it&#8217;s possible that your heart is inflamed and you should speak with your doctor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Platelet counts.<\/strong> Your platelet count may drop if you have elevated symptoms. Platelets may be checked by your healthcare professional both prior to and following Elevidys infusion.<\/p>\n\n\n\n<h2 id=\"what-types-of-interactions-can-happen\" class=\"wp-block-heading\">What Types of Interactions Can Happen?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Your healthcare professional will go over your vaccination history before beginning the corticosteroid medication prior to the Elevidys infusion. Because corticosteroids decrease the immune system, they may have an impact on how the body reacts to a vaccination. Any necessary vaccinations ought to be administered at least four weeks before corticosteroid medication is started.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The list of medications that could interact with Elevidys is not exhaustive. Inform your doctor or pharmacist about all of the prescription and over-the-counter (OTC) medications, vitamins, minerals, herbal remedies, and other supplements you currently use or have taken in the past. This will assist them in figuring out whether there are any interactions or whether your dosage needs to be changed.<\/p>\n\n\n\n<h2 id=\"frequently-asked-questions-about-elevidys\" class=\"wp-block-heading\">Frequently Asked Questions About Elevidys<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Some of the questions families are most curious about are how effective Elevidys gene therapy will be and how to reduce its costs. Here are some of the questions we receive from families:<\/p>\n\n\n<div id=\"rank-math-faq\" class=\"rank-math-block\">\n<div class=\"rank-math-list \">\n<div id=\"faq-question-1764153083449\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \">Could a child who has received Elevidys receive another (different) gene therapy in the future?<\/h3>\n<div class=\"rank-math-answer \">\n\n<p>When we examine the clinical studies of other gene therapies such as SGT-003 or RGX-202, we can see the phrase &#8216;not having received gene therapy before&#8217; among the exclusion criteria.<\/p>\n<p><strong>Sample exclusion criteria:<br \/><\/strong><br \/><strong>SGT-003<\/strong>: Current or prior treatment with an approved or investigational gene transfer drug.<\/p>\n<p><strong>RGX-202<\/strong>: Prior participation in a gene therapy trial OR recipient of a gene\u00a0therapy\u00a0drug.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1728832771702\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \">Is Sarepta aware that the cost of this gene therapy is not covered in many countries?<\/h3>\n<div class=\"rank-math-answer \">\n\n<p>As far as we know, the only country where gene therapy is currently covered by private health insurance is the USA. Will children of families living in other countries be expected to die?<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1728833081790\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \">Why is the cost of Elevidys treatment not reduced?<\/h3>\n<div class=\"rank-math-answer \">\n\n<p>Of course, every product will have a cost. The low number of children with Duchenne disease increases drug costs. We are aware of this! So will children of families who will never be able to afford these costs be left to die? I would like to ask this to the decision-makers in the pharmaceutical company!<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1728833604125\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \">Elevidys should review its pricing policy! Why is it so expensive?<\/h3>\n<div class=\"rank-math-answer \">\n\n<p>In some countries, purchasing power may be high or insurance companies may cover it. But what will families in countries with high inflation and low purchasing power do?<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1728833862440\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \">Sarepta must decide between money and the children&#8217;s lives?<\/h3>\n<div class=\"rank-math-answer \">\n\n<p>Sarepta must decide between money and children&#8217;s lives? Share data with countries, explain that gene therapy works, and create multiple payment options!<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1728832387311\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \">In which countries has Roche, which has the right to market the Elevidys gene therapy, applied for a licence?<\/h3>\n<div class=\"rank-math-answer \">\n\n<p>Roche obtains the exclusive right to launch and commercialize SRP-9001, Sarepta\u2019s micro-dystrophin gene therapy (ELEVIDYS) for Duchenne muscular dystrophy (DMD) outside the United States. Applications for approval are currently under review in, Switzerland, Singapore, Hong Kong and Saudi Arabia.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1739736490593\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \">Is the Roche Applying for Licenses for Gene Therapy in Every Country?<\/h3>\n<div class=\"rank-math-answer \">\n\n<p>We wrote to the company that developed the gene therapy (Sarepta) and the company that distributes it (Roche) many times! Do you know what their response was? They did not answer. [Read More: <a href=\"https:\/\/dmdwarrior.com\/duchenne-muscular-dystrophy-market-grows-but-not-all-families-have-access-to-treatments\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne Muscular Dystrophy Market Grows<\/a>]<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1728832580723\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \">Why is antibody testing only available in the United States?<\/h3>\n<div class=\"rank-math-answer \">\n\n<p>Is it not possible to make laboratory agreements for a simple antibody test? Is it fair to stress families out over antibody testing when gene therapy is so costly?<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1728833974425\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \">After receiving Elevidys gene therapy, if its effectiveness decreases or stops completely, can a second therapy be administered? Will the cost of this be covered free of charge?<\/h3>\n<div class=\"rank-math-answer \">\n\n<p>Families who can afford such a high cost or who have saved money through fundraising are most curious about this.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1761134853094\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \">Are Becker muscular dystrophy patients eligible for Elevidys gene therapy?<\/h3>\n<div class=\"rank-math-answer \">\n<img loading=\"lazy\" decoding=\"async\" width=\"1080\" height=\"1080\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2024\/12\/becker-eligible-elevidys.jpg\" class=\"alignright\" alt=\"Are patients with Becker muscular dystrophy eligible for Elevidys gene therapy\" title=\"\">\n<p>Becker Muscular Dystrophy (BMD) is a milder form of DMD with later onset and slower progression. It is also caused by mutations in the DMD gene, but those mutations result in retention of some function of the dystrophin protein. Becker Muscular dystrophy patients would likely not benefit from Elevidys gene therapy. &lt; <a href=\"https:\/\/dmdwarrior.com\/becker-muscular-dystrophy-eligible-for-elevidys-gene-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD Warrior<\/a> &gt;<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1728831673732\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \">If the Elevidys gene therapy doesn&#8217;t work, will Sarepta reimburse patients for the cost of the drug?<\/h3>\n<div class=\"rank-math-answer \">\n\n<p>An example of this was announced by Bluebird. The company will offer an up to 80% refund if the treatment, sold as Zynteglo, doesn\u2019t work as expected. [<strong>Source:<\/strong> <a href=\"https:\/\/investor.bluebirdbio.com\/news-releases\/news-release-details\/bluebird-bio-announces-us-commercial-infrastructure-enable\" target=\"_blank\" rel=\"noreferrer noopener\">BlueBird<\/a>]<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1741357550389\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \">Who Should Not Use Elevidys?<\/h3>\n<div class=\"rank-math-answer \">\n\n<p>Any deletion in exon 8 and\/or exon 9 in the DMD gene, should not receive ELEVIDYS.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1761047506785\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \">Why Was Elevidys Not Approved by the European Medicines Agency (EMA)?<\/h3>\n<div class=\"rank-math-answer \">\n<img loading=\"lazy\" decoding=\"async\" width=\"1080\" height=\"1080\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2024\/12\/elevidys-NOT-approved-ema.jpg\" class=\"alignright\" alt=\"Why Elevidys Was Not Approved by the European Medicines Agency (EMA)\" title=\"\">\n<p><strong>Why didn&#8217;t EMA approve of Elevidys?<\/strong> In the pivotal study (the main evidence submitted), there was no statistically significant improvement in motor\u2011function (as measured using the North Star Ambulatory Assessment, NSAA) at 12\u202fmonths between the Elevidys arm and placebo arm. The difference was only 0.65 points on a 34\u2011point scale, which the EMA concluded \u201cmay be due to chance\u201d. (<a href=\"https:\/\/dmdwarrior.com\/why-elevidys-was-not-approved-by-the-european-medicines-agency-ema\/\">Read More<\/a>)<\/p>\n\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n\n\n<p class=\"wp-block-paragraph\"><strong>Read More<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/why-elevidys-was-not-approved-by-the-european-medicines-agency-ema\/\" target=\"_blank\" rel=\"noreferrer noopener\">Why Elevidys Was Not Approved by the European Medicines Agency (EMA)?<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Duchenne muscular dystrophy (DMD) is a genetic condition that causes progressive muscle weakness. Boys are more likely to develop DMD, with symptoms often appearing in early childhood. DMD has no known cure. Medical professionals use physical therapy, steroids, and assistive technologies such as wheelchairs and braces to manage symptoms. New medications targeting the underlying genetic [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":8418,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[45],"tags":[121,66,33,61,65],"class_list":["post-1497","post","type-post","status-publish","format-standard","has-post-thumbnail","category-approved-therapies-for-duchenne-muscular-dystrophy","tag-delandistrogene-moxeparvovec","tag-duchenne-muscular-dystrophy","tag-elevidys","tag-gene-therapy","tag-sarepta"],"subtitle":"La terapia g\u00e9nica para la distrofia muscular de Duchenne (DMD), ELEVIDYS, de Sarepta, ha sido aprobada en Estados Unidos, Emiratos \u00c1rabes Unidos, Catar, Kuwait, Bar\u00e9in, Om\u00e1n, Brasil, Israel y Jap\u00f3n. Sin embargo, con un precio de 1.173.000 yuanes, resulta muy dif\u00edcil de acceder para muchas familias. En este art\u00edculo, respondemos a las preguntas m\u00e1s frecuentes sobre la primera terapia g\u00e9nica aprobada para la distrofia muscular de Duchenne.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts\/1497","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/comments?post=1497"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts\/1497\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/media\/8418"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/media?parent=1497"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/categories?post=1497"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/tags?post=1497"}],"curies":[{"name":"gracias","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}