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Robert F. Kennedy Jr. wants health agencies to use a lot more AI. After the MAHA report, experts have some concerns

Robert F. Kennedy Jr. wants health agencies to use a lot more AI. After the MAHA report, experts have some concerns

Boston Globea day ago

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And with the apparent inclusion of material imagined by
AI, referred to as hallucinations, in the 'Make America Healthy Again' report, they see an ominous sign for the administration's ability to deploy it safely.
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'If they were proceeding more apace, and I'm not talking about glacial government pace, I'm talking about responsible pace, this wouldn't happen,' said Oren Etzioni, a professor emeritus at the University of Washington and entrepreneur who studies AI. 'The presence of these embarrassing missteps just shows that it's amateur hour.'
Kennedy has expressed ambitious but vague plans, usually in the context of cutting costs. He has outlined his vision during several congressional hearings, including one to replace the use of animals in experimental testing and some steps in clinical trials.
'We're phasing out most animal studies . . . because we can accomplish a lot of those goals on safety and efficacy with AI technology,' Kennedy said.
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He also mentioned using AI to analyze data that HHS and other agencies have collected on patients, such as people on Medicare and Medicaid. He said they've recruited experts to 'transform our agency for a central hub for AI.'
So far, the Food and Drug Administration has announced
computer and AI modeling.
'In the long-term (3-5 years), FDA will aim to make animal studies the exception rather than the norm for pre-clinical safety/toxicity testing,' the FDA said in its road map.
But the mistakes in the 'Make America Healthy Again' report have experts skeptical of HHS's ability to use AI correctly. The report
cited
sources that do not exist and had garbled footnotes,
The agency declined to answer definitively whether AI was used on the report and why it contained nonexistent citations, but rather only highlighted the substance of the report. It also did not offer specifics about protocols for responsible AI use.
'HHS is addressing the risk of AI-generated errors through rigorous validation, human oversight, and strict quality controls,' a spokesperson said in a statement. 'AI tools are designed to support — not replace — expert judgment.'
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Experts say the specifics of how AI is implemented will bethe true measure of whether the efforts at HHS will succeed or end up being harmful.
'I'm actually deeply optimistic about what [AI] can do in a lot of areas, including the ones that the secretary mentioned,' said Ziad Obermeyer, a physician and researcher at the University of California Berkeley who studies AI in biomedicine. 'What my research has shown is that it actually comes down to some of the really boring details that make the difference between a good, powerful algorithm that helps people, and one that really messes things up.'
Republicans who work on AI issues in the Senate supported Kennedy's goals but also agreed on the importance of rolling it out with the right protections.
'This is going to be the future,' said Indiana Senator Todd Young. 'I mean, we'd be doing something wrong if, if the head of our health agency wasn't talking about using AI.'
The two general use cases that Kennedy has mentioned, replacing steps in clinical trials and analyzing patient data, have some potential issues in common, including that AI can generate false information.
But they also have risks unique to each case. Privacy, for example, is a serious concern with patient data. If not properly stripped of identifying factors, even supposedly anonymized data can be re-identified, as has happened in some cases.
'The most secret private information that people have is their health care data, and so AI should not be used in any way that does not have the strongest possible safeguards,' said Massachusetts Senator Ed Markey, a Democrat. 'We could have an absolute privacy catastrophe.'
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Harvard Law School professor I. Glenn Cohen, who studies medical ethics and AI, said that the idea holds great potential, but that the administration would need to be very transparent about how it is protecting data and would be wise to run smaller pilot studies first.
'The 'move fast and break things' ethos of Silicon Valley may be appropriate in some parts of life — I don't really care if you're doing it for the order of Instagram postings," Cohen said. 'But it's not a philosophy we advocate for physicians or an attitude I think most people want health care to take.'
The key limitation of AI is that it is only as good as the dataset used to build it. In specific areas where scientific data is really good and outcomes are predictable, such as in the structure of proteins, scientists have built powerful AI tools. AI can also help doctors and patients assess symptoms.
But those are different from the discovery of new information, experts say, which is what a lot of science and clinical trials for novel treatments are designed to explore.
Allison Coffin is a researcher at Creighton University who studies hearing loss, including that caused by certain medicines. She uses mostly zebra fish in her work, but also rodents. She says her lab is working on AI tools to help identify potential toxins in order to conduct more targeted research. But, she said, AI would always be used as an idea generator for testing in animals, not to replace them.
'That's an excellent case for AI, because AI can rapidly assess millions of potential drug structures. But you would still want to test their efficacy for new therapies in an animal,' Coffin said. 'I would never want to take a medication that hadn't been given to a living creature before, and I would think most people wouldn't. Do we want to be the first to take medication because a computer model says that it's safe?'
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Other scientists questioned the ability of the government to do the cutting-edge research necessary after the administration's deep cuts to research funding and staff.
'Honestly I'm struggling for what to say,' wrote Sean Eddy, a Harvard scientist who works on building computer models for biology and genomic research. 'I just don't see how it makes sense for HHS to talk about delivering innovative technological breakthroughs while they're destabilizing and belittling the US scientific research enterprise. . . . Every lab at Harvard that does this kind of research, including my own, has had all their federal funding terminated.'
Experts also question whether Kennedy understands where AI technology actually is today versus its potential capacities. Many cited cautionary tales of much lower-stakes AI deployment gone wrong, such as companies that
'Doing things like simulating an entire body in order to save clinical trials is just grossly unrealistic where we sit right now,' said Gary Marcus, a professor emeritus at New York University and critic of AI enthusiasm. 'If we're lucky, we can do it in 40 [years], but we certainly can't now. That's just a pipe dream.'
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Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne
Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne

Associated Press

timean hour ago

  • Associated Press

Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun 15, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety update regarding ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy, and steps the Company is taking to strengthen the safety profile in non-ambulatory patients. These steps follow a second reported case of acute liver failure (ALF) resulting in death. The cases of ALF to date have both occurred in non-ambulatory individuals with Duchenne. Sarepta extends its deepest sympathies to the affected families and care teams. Key Safety Initiatives Evaluating and Enhancing Immunosuppressive Regimen: As part of a comprehensive review of safety data, Sarepta is taking proactive steps to mitigate the risk of acute liver failure in non-ambulatory patients. Sarepta is working to immediately convene an independent group of leading experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for ELEVIDYS. This panel will evaluate data and assess our proposed regimen, which includes sirolimus and is supported by preclinical data demonstrating the effectiveness of additional immunosuppression in moderating liver enzyme elevations, a key factor in mitigating potential safety events. Sarepta will share the panel's recommendations with the U.S. Food & Drug Administration (FDA), and implementation of any new regimen will be subject to FDA guidance and allowance. Suspending Shipments of ELEVIDYS for Non-Ambulatory Patients: Sarepta is temporarily suspending shipments of ELEVIDYS for non-ambulatory patients while an enhanced immunosuppressive regimen is evaluated, discussed with regulatory bodies, and put in place. For ambulatory patients, no treatment changes are being proposed and the current practice of administering corticosteroids before and after ELEVIDYS infusion, along with post-treatment monitoring, remains the same. ENVISION Study Paused: Sarepta has voluntarily paused dosing in the ENVISION clinical study (also known as Study SRP-9001-303). FDA concurs with this action. The pause will allow for the evaluation of a protocol amendment to incorporate an enhanced immunosuppressive regimen for the non-ambulatory patient cohort and incorporate any additional feedback from the FDA. Regulatory alignment is needed before screening and dosing in ENVISION may resume. ENVISION is a global, randomized, double-blind, placebo-controlled trial evaluating ELEVIDYS in older ambulatory and non-ambulatory individuals living with Duchenne muscular dystrophy. In the U.S., it serves as the confirmatory trial required under the FDA's accelerated approval pathway for non-ambulatory patients. 'Our paramount priority is the safety and well-being of the patients we serve. We are taking immediate, decisive steps to better understand and mitigate the risk of acute liver failure, including enhancing the immunosuppressive regimen, for those with Duchenne who are non-ambulatory,' said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research & development, Sarepta. 'We are deeply saddened by the loss of a second patient and extend our heartfelt condolences to the patient's family and his care team during this incredibly difficult time. Duchenne muscular dystrophy is a devastating disease that profoundly affects lives and often cuts them far too short. With more than 900 individuals treated to-date, we know how much hope families place in new treatment options like ELEVIDYS – and we are committed to honoring that hope by acting swiftly, guided by scientific rigor and the insights of leading experts, to strengthen safety for all future patients.' Commitment to Long-Term Safety and Understanding Sarepta remains committed to a thorough approach and the highest standards of patient safety and scientific rigor. The event has been reported to FDA and global health authorities and will inform ongoing discussions around a potential label update to reflect the risk of severe ALF and additional immune management strategies for non-ambulatory patients. While elevated liver enzymes are a known class effect of all AAV-based gene therapies, the exact mechanism behind AAV-related liver toxicity remains unclear. Current evidence suggests it is likely driven by an adaptive immune response. The Company will provide additional updates as appropriate. Investor Conference Call Details Sarepta will be hosting a conference call and webcast to discuss this update and provide an update on the Company's business on Monday, June 16, 2025, at 8:00 am Eastern time. The event will be webcast live under the investor relations section of Sarepta's website at: and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone. About ELEVIDYS (delandistrogene moxeparvovec-rokl) ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). IMPORTANT SAFETY INFORMATION CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: Infusion-related Reactions: Acute Serious Liver Injury: Immune-mediated Myositis: Myocarditis: Preexisting Immunity against AAVrh74: Adverse Reactions: Report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). For further information, please see the full Prescribing Information. About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit or follow us on LinkedIn, X, Instagram and Facebook. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website We encourage investors and potential investors to consult our website regularly for important information about us. Forward-Looking Statements This statement contains 'forward-looking statements.' Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as 'believe,' 'anticipate,' 'plan,' 'expect,' 'will,' 'may,' 'intend,' 'prepare,' 'look,' 'potential,' 'possible' and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, research and development programs, clinical trials, ELEVIDYS, the potential benefits of an enhanced immunosuppression regimen in dosing in non-ambulatory patients, and expected plans and milestones, including providing additional updates as appropriate and engaging with regulators on an enhanced immunosuppressive regimen for dosing in non-ambulatory patients. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials are positive, these data may not be sufficient to support approval by the FDA or other global regulatory authorities; success in clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or with advisory committee recommendations, or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; we may not be able to comply with all FDA requests in a timely manner or at all; the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business; and those risks identified under the heading 'Risk Factors' in our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to source version on CONTACT: Investor Contact: Ian Estepan 617-274-4052 [email protected] Contacts: Tracy Sorrentino 617-301-8566 [email protected] Hoeger 617-710-3898 [email protected] KEYWORD: UNITED STATES NORTH AMERICA MASSACHUSETTS INDUSTRY KEYWORD: INFECTIOUS DISEASES HEALTH GENETICS PHARMACEUTICAL CLINICAL TRIALS SOURCE: Sarepta Therapeutics, Inc. Copyright Business Wire 2025. PUB: 06/15/2025 01:00 AM/DISC: 06/15/2025 01:01 AM

Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne
Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne

Business Wire

time4 hours ago

  • Business Wire

Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety update regarding ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy, and steps the Company is taking to strengthen the safety profile in non-ambulatory patients. These steps follow a second reported case of acute liver failure (ALF) resulting in death. The cases of ALF to date have both occurred in non-ambulatory individuals with Duchenne. Sarepta extends its deepest sympathies to the affected families and care teams. Key Safety Initiatives Evaluating and Enhancing Immunosuppressive Regimen: As part of a comprehensive review of safety data, Sarepta is taking proactive steps to mitigate the risk of acute liver failure in non-ambulatory patients. Sarepta is working to immediately convene an independent group of leading experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for ELEVIDYS. This panel will evaluate data and assess our proposed regimen, which includes sirolimus and is supported by preclinical data demonstrating the effectiveness of additional immunosuppression in moderating liver enzyme elevations, a key factor in mitigating potential safety events. Sarepta will share the panel's recommendations with the U.S. Food & Drug Administration (FDA), and implementation of any new regimen will be subject to FDA guidance and allowance. Suspending Shipments of ELEVIDYS for Non-Ambulatory Patients: Sarepta is temporarily suspending shipments of ELEVIDYS for non-ambulatory patients while an enhanced immunosuppressive regimen is evaluated, discussed with regulatory bodies, and put in place. For ambulatory patients, no treatment changes are being proposed and the current practice of administering corticosteroids before and after ELEVIDYS infusion, along with post-treatment monitoring, remains the same. ENVISION Study Paused: Sarepta has voluntarily paused dosing in the ENVISION clinical study (also known as Study SRP-9001-303). FDA concurs with this action. The pause will allow for the evaluation of a protocol amendment to incorporate an enhanced immunosuppressive regimen for the non-ambulatory patient cohort and incorporate any additional feedback from the FDA. Regulatory alignment is needed before screening and dosing in ENVISION may resume. ENVISION is a global, randomized, double-blind, placebo-controlled trial evaluating ELEVIDYS in older ambulatory and non-ambulatory individuals living with Duchenne muscular dystrophy. In the U.S., it serves as the confirmatory trial required under the FDA's accelerated approval pathway for non-ambulatory patients. 'Our paramount priority is the safety and well-being of the patients we serve. We are taking immediate, decisive steps to better understand and mitigate the risk of acute liver failure, including enhancing the immunosuppressive regimen, for those with Duchenne who are non-ambulatory,' said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research & development, Sarepta. 'We are deeply saddened by the loss of a second patient and extend our heartfelt condolences to the patient's family and his care team during this incredibly difficult time. Duchenne muscular dystrophy is a devastating disease that profoundly affects lives and often cuts them far too short. With more than 900 individuals treated to-date, we know how much hope families place in new treatment options like ELEVIDYS – and we are committed to honoring that hope by acting swiftly, guided by scientific rigor and the insights of leading experts, to strengthen safety for all future patients.' Commitment to Long-Term Safety and Understanding Sarepta remains committed to a thorough approach and the highest standards of patient safety and scientific rigor. The event has been reported to FDA and global health authorities and will inform ongoing discussions around a potential label update to reflect the risk of severe ALF and additional immune management strategies for non-ambulatory patients. While elevated liver enzymes are a known class effect of all AAV-based gene therapies, the exact mechanism behind AAV-related liver toxicity remains unclear. Current evidence suggests it is likely driven by an adaptive immune response. The Company will provide additional updates as appropriate. Investor Conference Call Details Sarepta will be hosting a conference call and webcast to discuss this update and provide an update on the Company's business on Monday, June 16, 2025, at 8:00 am Eastern time. The event will be webcast live under the investor relations section of Sarepta's website at: and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone. About ELEVIDYS (delandistrogene moxeparvovec-rokl) ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). IMPORTANT SAFETY INFORMATION CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: Infusion-related Reactions: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate. ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available. Discontinue infusion for anaphylaxis. Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks. Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels). Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended. Immune-mediated Myositis: In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction. Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient's clinical presentation and medical history if these symptoms occur. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath. Advise patients to contact a physician immediately if they experience cardiac symptoms. Preexisting Immunity against AAVrh74: In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies. Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia. Report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). For further information, please see the full Prescribing Information. About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit or follow us on LinkedIn, X, Instagram and Facebook. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at We encourage investors and potential investors to consult our website regularly for important information about us. Forward-Looking Statements This statement contains 'forward-looking statements.' Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as 'believe,' 'anticipate,' 'plan,' 'expect,' 'will,' 'may,' 'intend,' 'prepare,' 'look,' 'potential,' 'possible' and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, research and development programs, clinical trials, ELEVIDYS, the potential benefits of an enhanced immunosuppression regimen in dosing in non-ambulatory patients, and expected plans and milestones, including providing additional updates as appropriate and engaging with regulators on an enhanced immunosuppressive regimen for dosing in non-ambulatory patients. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials are positive, these data may not be sufficient to support approval by the FDA or other global regulatory authorities; success in clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or with advisory committee recommendations, or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; we may not be able to comply with all FDA requests in a timely manner or at all; the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business; and those risks identified under the heading 'Risk Factors' in our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review. Any of the foregoing risks could materially and adversely affect the Company's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained herein. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law.

Medicaid enrollees fear losing health coverage if Congress enacts work requirements

time4 hours ago

Medicaid enrollees fear losing health coverage if Congress enacts work requirements

It took Crystal Strickland years to qualify for Medicaid, which she needs for a heart condition. Strickland, who's unable to work due to her condition, chafed when she learned that the U.S. House has passed a bill that would impose a work requirement for many able-bodied people to get health insurance coverage through the low-cost, government-run plan for lower-income people. 'What sense does that make?' she asked. 'What about the people who can't work but can't afford a doctor?' The measure is part of the version of President Donald Trump's 'Big Beautiful' bill that cleared the House last month and is now up for consideration in the Senate. Trump is seeking to have it passed by July 4. The bill as it stands would cut taxes and government spending — and also upend portions of the nation's social safety net. For proponents, the ideas behind the work requirement are simple: Crack down on fraud and stand on the principle that taxpayer-provided health coverage isn't for those who can work but aren't. The measure includes exceptions for those who are under 19 or over 64, those with disabilities, pregnant women, main caregivers for young children, people recently released from prisons or jails — or during certain emergencies. It would apply only to adults who receive Medicaid through expansions that 40 states chose to undertake as part of the 2010 health insurance overhaul. Many details of how the changes would work would be developed later, leaving several unknowns and causing anxiety among recipients who worry that their illnesses might not be enough to exempt them. Advocates and sick and disabled enrollees worry — based largely on their past experience — that even those who might be exempted from work requirements under the law could still lose benefits because of increased or hard-to-meet paperwork mandates. Strickland, a 44-year-old former server, cook and construction worker who lives in Fairmont, North Carolina, said she could not afford to go to a doctor for years because she wasn't able to work. She finally received a letter this month saying she would receive Medicaid coverage, she said. 'It's already kind of tough to get on Medicaid,' said Strickland, who has lived in a tent and times and subsisted on nonperishable food thrown out by stores. 'If they make it harder to get on, they're not going to be helping.' Steve Furman is concerned that his 43-year-old son, who has autism, could lose coverage. The bill the House adopted would require Medicaid enrollees to show that they work, volunteer or go to school at least 80 hours a month to continue to qualify. A disability exception would likely apply to Furman's son, who previously worked in an eyeglasses plant in Illinois for 15 years despite behavioral issues that may have gotten him fired elsewhere. Furman said government bureaucracies are already impossible for his son to navigate, even with help. It took him a year to help get his son onto Arizona's Medicaid system when they moved to Scottsdale in 2022, and it took time to set up food benefits. But he and his wife, who are retired, say they don't have the means to support his son fully. 'Should I expect the government to take care of him?' he asked. 'I don't know, but I do expect them to have humanity.' About 71 million adults are enrolled in Medicaid now. And most of them — around 92% — are working, caregiving, attending school or disabled. Earlier estimates of the budget bill from the Congressional Budget Office found that about 5 million people stand to lose coverage. A KFF tracking poll conducted in May found that the enrollees come from across the political spectrum. About one-fourth are Republicans; roughly one-third are Democrats. The poll found that about 7 in 10 adults are worried that federal spending reductions on Medicaid will lead to more uninsured people and would strain health care providers in their area. About half said they were worried reductions would hurt the ability of them or their family to get and pay for health care. Amaya Diana, an analyst at KFF, points to work requirements launched in Arkansas and Georgia as keeping people off Medicaid without increasing employment. Amber Bellazaire, a policy analyst at the Michigan League for Public Policy, said the process to verify that Medicaid enrollees meet the work requirements could be a key reason people would be denied or lose eligibility. 'Massive coverage losses just due to an administrative burden rather than ineligibility is a significant concern,' she said. One KFF poll respondent, Virginia Bell, a retiree in Starkville, Mississippi, said she's seen sick family members struggle to get onto Medicaid, including one who died recently without coverage. She said she doesn't mind a work requirement for those who are able — but worries about how that would be sorted out. 'It's kind of hard to determine who needs it and who doesn't need it,' she said. Lexy Mealing, 54 of Westbury, New York, who was first diagnosed with breast cancer in 2021 and underwent a double mastectomy and reconstruction surgeries, said she fears she may lose the medical benefits she has come to rely on, though people with 'serious or complex' medical conditions could be granted exceptions. She now works about 15 hours a week in 'gig' jobs but isn't sure she can work more as she deals with the physical and mental toll of the cancer. Mealing, who used to work as a medical receptionist in a pediatric neurosurgeon's office before her diagnosis and now volunteers for the American Cancer Society, went on Medicaid after going on short-term disability. 'I can't even imagine going through treatments right now and surgeries and the uncertainty of just not being able to work and not have health insurance,' she said. Felix White, who has Type I diabetes, first qualified for Medicaid after losing his job as a computer programmer several years ago. The Oreland, Pennsylvania, man has been looking for a job, but finds that at 61, it's hard to land one. Medicaid, meanwhile, pays for a continuous glucose monitor and insulin and funded foot surgeries last year, including one that kept him in the hospital for 12 days. 'There's no way I could have afforded that,' he said. 'I would have lost my foot and probably died.'

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