anxa11 suppression restores muscular function in the mdx mouse model of duchenne muscular dystrophy dmd

Original title: “ANXA11 suppression restores muscular function in the mdx mouse model of Duchenne muscular dystrophy (DMD).”

advancements from the evolve study for assessing real world experience with eteplirsen golodirsen and casimersen for the treatment of dmd

Original title: “Advancements from the EVOLVE study for assessing real-world experience with eteplirsen, golodirsen and casimersen for the treatment of DMD.”

cost utility analysis of the treatment with ataluren plus standard of care compared with standard of care alone in patients with duchenne muscular dystrophy in brazil

Original title: “Cost-Utility Analysis of the Treatment With Ataluren Plus Standard of Care Compared With Standard of Care Alone in Patients With Duchenne Muscular Dystrophy in Brazil.”

Sarepta Therapeutics says the FDA has agreed to review a regulatory package for Amondys 45 and Vyondys 53 after they failed a confirmatory trial, but whether the agency will agree to approve them is still unknown.

Original title: “Sarepta Plans FDA Run for Duchenne Exon Skippers Despite Confirmatory Trial Failure”

economic burden of duchenne muscular dystrophy from a societal perspective in mumbai india

Original title: “Economic burden of Duchenne muscular dystrophy from a societal perspective in Mumbai, India.”

Solid Biosciences’ SGT-003 is the only late‑stage program to show early cardiac benefit across biomarkers and function, according to William Blair.

Original title: “Solid Adds to Differentiated Profile With New Data for Duchenne Gene Therapy”

The biopharma business model is misaligned with rare diseases, and children are paying the price. Home » Rare Diseases » Rethinking How Rare Disease Treatments Get to Patients ... The biopharma business model is misaligned with rare diseases, and children are paying the price. Recent policy and market changes have sparked a dramatic shift in investment toward therapies with broad commercial demand — leaving hundreds of rare disease programs shelved, often despite promising clinical data. Matthews is a testament to how awareness can inspire progress. “When I joined forces with CureDuchenne, this was a relatively unknown disease,” he says. “But we’re at the point now where there are human clinical trials, and the symptoms of this disease are being checked.” “There have been huge leaps forward in genetic medicines to treat rare diseases, but the business model to support their advancement hasn’t evolved at the same pace,” shared Craig Martin, who founded the Orphan Therapeutics Accelerator (OTXL) to reimagine how rare disease treatments are financed and advanced. As a nonprofit, OTXL can incentivize companies to donate programs and negotiate deferred or reduced development costs with industry partners. In partnership with the American Society for Gene and Cell Therapy, OTXL is also building an AI-powered marketplace to surface shelved assets and match them with aligned funders, opening the door to family offices, impact investors, and others historically excluded from this space. Clay Matthews, the Super Bowl-winning linebacker for the Green Bay Packers, became a part of the rare disease community almost by chance. His first exposure to Duchenne, a rare muscle degeneration disease that affects boys, was for a CureDuchenne PSA that he lent his likeness to.

Original title: “Rethinking How Rare Disease Treatments Get to Patients - Future of Personal Health”

Data presented at the 2026 Muscular Dystrophy Association meeting could have readthroughs to companies developing therapies for spinal muscular atrophy, Duchenne muscular dystrophy and Becker muscular dystrophy.

Original title: “MDA 2026: Biogen, Sarepta, Edgewise Address Muscle Disorders”

Capricor Therapeutics’ deramiocel was rejected in July 2025, potentially caught between Nicole Verdun, a former top biologics regulator at the FDA, and Vinay Prasad, director of the Center for Biologics Evaluation and Research.

Original title: “Capricor Shares Rise as FDA Lifts Duchenne Cell Therapy Rejection”

Xenon shares skyrocketed nearly 50% on results that handily beat Wall Street expectations. Elsewhere, Capricor got a decision date for a spurned cell therapy and AbbVie and Regeneron showcased obesity data.

Original title: “Xenon hits a ‘home run’ in epilepsy; FDA restarts review of Duchenne cell therapy”

Dyne Therapeutics is plotting an approval application for z-rostudirsen in the back half of 2026—a push that will only be bolstered by the departure of controversial CBER chief Vinay Prasad, according to analysts at Stifel.

Original title: “Dyne Plans Post-Prasad FDA Run as Duchenne Exon Skipper Sustains Benefit in Long Term Data”

Discover how the UAE is leading the way in precision medicine, offering hope to rare disease patients through advanced genomics and personalized care. Learn about the transformative impact of early genomic screening and targeted therapies. In disorders such as spinal muscular atrophy or duchenne muscular dystrophy, therapies like gene replacement, exon-skipping, and molecular modulation are designed to correct or compensate for specific genetic defects. Without precise genetic characterisation, these treatments cannot be appropriately offered,” Dr. Vivek Mundada, consultant paediatric neurology at Medcare Royal Speciality Hospital, told Gulf News. “Moreover, rare diseases frequently involve multiple organ systems. One of the most significant findings in rare disease care has been the growing accessibility of early genomic screening. Advanced sequencing technologies can now analyse vast portions of the genome in a matter of days, a process that once took years. “Early genomic screening is also transforming patient journeys. Instead of years of sequential testing, whole-exome or whole-genome sequencing can provide answers early in life. When genomic data is integrated with clinical records, imaging, and digital pathology, diagnostic time is significantly reduced. Meanwhile, health experts have highlighted the UAE’s role as a regional leader in advanced healthcare, especially in genetics and rare diseases. According to Mundada, “What distinguishes the UAE is its ability to scale innovation quickly. When supported by structured policy and digital integration, precision medicine does not remain confined to academic centres, it becomes part of national healthcare delivery.” · “The UAE is demonstrating how personalised medicine can move from concept to clinical reality, particularly for patients living with rare diseases,” added Thomas. By analysing a patient’s genetic makeup, doctors can determine the underlying cause of a condition more accurately and, in some cases, identify targeted therapies. “Precision medicine allows us to move beyond symptom-based treatment to gene-level diagnosis and targeted intervention.

Original title: “How precision medicine revolutionises rare disease treatment in UAE”

FDA delays treatments for rare diseases, risking lives of children with conditions like DMD and ALS. Trump signed the Right to Try Act in 2018, which allows those with life-threatening diseases or conditions to access experimental drugs that have completed at least Phase 1 clinical trials but have not yet received full FDA approval. There is bipartisan support to speed up this process; Sen. Kirsten Gillibrand (D-NY) sounded as concerned as the Republican members of the committee. ... Maybe U.S. Secretary of Health and Human Services Robert F. Kennedy Jr., who has pushed for faster drug approvals in areas like rare diseases, can apply some pressure. Campbell offered several solutions, and urged Congress to pass the Biomanufacturing Excellence Act of 2025 (H.R. 6089 and S. 3188), which will “advance manufacturing methods to ensure innovative products can move rapidly from clinical to commercial scale.” · Cara O’Neill, Chief Science Officer & Co-Founder of Cure Sanfilippo Foundation and the mother of a 16-year-old daughter with Sanfilippo Syndrome, told the committee how she watched a young girl die of the disease, which is a form of childhood dementia. However, her own daughter was able to receive treatment during a trial and is thriving. Angelina Olivera, the mother of a 14-year-old son who has DMD, who also watched her brothers die at ages 20 and 22 of the disease, told me there is only one promising new treatment for DMD but the FDA won’t approve it for her son Ryu. A study was conducted on 1,200 patients using Elevidys from Sarepta Therapeutics and revealed promising results. The gene therapy slowed disease progression by 70-73 percent compared to untreated children. Elevidys was approved for a few types of patients, but since a couple of patients in the trial treatment died of acute liver failure afterwards, the FDA halted access to those in wheelchairs like her son, who lost his ability to walk at age 9. Sarepta Therapeutics said those severe outcomes were not the result of the treatment. The two DMD patients who died were in advanced stages, and a third fatality occurred in a separate trial of a higher-dose gene therapy for a different muscle disease.

Original title: “FDA Cruelly Holding Up Approval of Treatments for Rare Diseases, Despite Children Likely to Die Soon”

After a rocky 2025, Sarepta Therapeutics’ executives admit they have work to do to bring patients back into the fold as sales of Duchenne muscular dystrophy gene therapy Elevidys continue to decline.

Original title: “After Sarepta’s Annus Horribilis, Elevidys Sales Expected To Continue Downward Spiral”

Sarepta to present new long-term ... slowing disease progression and supporting informed treatment decisions. ... U.S. Recommended Uniform Screening Panel (RUSP): The inclusion of Duchenne on the RUSP represents a pivotal advancement for the Duchenne community, encouraging broader newborn screening at the state level and empowering more families with early diagnosis and timely information to pursue earlier care, including access to available therapies and clinical trials... Sarepta to present new long-term and safety data across gene therapy and exon-skipping programs at 2026 Muscular Dystrophy Association Clinical & Scientific Conference: Sarepta will present new clinical and real‑world evidence at MDA 2026 showing long‑term efficacy, safety, and caregiver‑reported impact of its gene therapy and exon‑skipping treatments for Duchenne muscular dystrophy, reinforcing dystrophin’s role in slowing disease progression and supporting informed treatment decisions. ... U.S. Recommended Uniform Screening Panel (RUSP): The inclusion of Duchenne on the RUSP represents a pivotal advancement for the Duchenne community, encouraging broader newborn screening at the state level and empowering more families with early diagnosis and timely information to pursue earlier care, including access to available therapies and clinical trials. Net product revenue reached $1.86B in 2025 as Sarepta posted a $713M GAAP net loss but exited the year with $954M cash and expects 2026 profitability. At the same time, our PMO exon-skipping therapies continue to demonstrate durable clinical value, exceptional safety, extraordinary real‑world outcomes, and unwavering support from families and physicians. Finally, with five clinical‑stage RNAi programs and multiple readouts ahead, we are poised for meaningful growth while advancing therapies that can profoundly change the lives of patients living with rare disease.” Feb 4, 2026 Sarepta Therapeutics Announces Approval of Clinical Trial Application for SRP-1005, Its Investigational Treatment for Huntington’s Disease

Original title: “Inside Sarepta’s tumultuous $2.2B year in Duchenne gene therapy”

The “Omirge Sen” Foundation was established to support families whose members suffer from hereditary neuromuscular diseases, including myopathies (Duchenne/Becker), spinal muscular atrophy (SMA), congenital myasthenia,… Continue reading Stories of Our Little Heroes The “Omirge Sen” Foundation was established to support families whose members suffer from hereditary neuromuscular diseases, including myopathies (Duchenne/Becker), spinal muscular atrophy (SMA), congenital myasthenia, Charcot-Marie syndrome, and others. This video was filmed featuring our beneficiaries for Rare Disease Day to raise awareness of Duchenne muscular dystrophy.

Original title: “Stories of Our Little Heroes - Rare Disease Day 2026”

The “Omirge Sen” Foundation was established to support families whose members suffer from hereditary neuromuscular diseases, including myopathies (Duchenne/Becker), spinal muscular atrophy (SMA), congenital myasthenia,… Continue reading Story of our little heroes The “Omirge Sen” Foundation was established to support families whose members suffer from hereditary neuromuscular diseases, including myopathies (Duchenne/Becker), spinal muscular atrophy (SMA), congenital myasthenia, Charcot-Marie syndrome, and others. This video was filmed featuring our beneficiaries for Rare Disease Day to raise awareness of Duchenne muscular dystrophy.

Original title: “Story of our little heroes - Rare Disease Day 2026”