Top 10 muscular dystrophy news stories of 2025

Throughout 2025, Muscular Dystrophy News Today brought you daily coverage of the latest muscular dystrophy (MD)-related clinical research and scientific breakthroughs.

Here are the year’s top 10 most-read stories we published last year, each with a brief description.

No. 10 — FSHD patients sought for first EPI-321 clinical trial

Enrollment has begun for a first-in-human Phase 1/2 trial of EPI-321, an experimental epigenetic therapy for facioscapulohumeral muscular dystrophy (FSHD). The study will enroll nine adults ages 18 to 75, with each receiving a single infusion at one of two dose levels. Its primary goal is to assess safety. Recruitment is underway in the U.S. and New Zealand, with an Australian site planned. The first participant has been dosed, and initial results are expected early this year. EPI-321 aims to switch off the faulty DUX4 gene, which is normally inactive in healthy muscle cells. In FSHD, this switch is dysfunctional, keeping the gene active and producing a protein that damages muscle cells.

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No. 9 — New redosable DMD gene therapy gets funding boost

CureDuchenne is investing $1 million in Entos Pharmaceuticals‘ development of a new gene therapy for Duchenne muscular dystrophy (DMD) that addresses the limitations of current gene therapies. Existing viral-based gene therapies, which deliver a shortened version of the DMD gene to replace the defective gene that causes DMD, are typically given only once. The new approach aims to provide the full-length DMD gene and enable repeat dosing, potentially expanding eligibility and effectiveness. Entos’ Fusogenix PLV platform delivers therapeutic genes by encapsulating them in specialized membranes that fuse with a patient’s cells, allowing the genes to enter the cells directly.

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No. 8 — New way to improve DMD treatments developed

Researchers have identified a way to improve exon-skipping treatments for DMD by enabling the therapy to reach muscle cells more effectively. The DMD gene consists of segments called exons that are spliced together to produce the dystrophin protein. Exon-skipping therapies remove specific exons, allowing cells to make a shorter but functional protein. Several are approved in the U.S., and they use phosphorodiamidate morpholino oligomers, or PMOs. Still, their effectiveness is limited because inflammation and scarring in DMD muscles impede the treatment’s entry into muscle cells. The team used a second PMO to reduce inflammation and scarring, thereby making muscle cells more accessible and enabling exon-skipping therapies to be more effective.

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No. 7 — SAT-3247 shows early signs of efficacy in DMD

Satellos Bioscience‘s oral drug SAT-3247 was found to be safe and well-tolerated in a Phase 1 study and showed early signs of improving muscle strength in a small study of adults with DMD. The therapy is designed to boost muscle stem cell activity to support muscle repair. It works independently of dystrophin, allowing it to be used alone or with other treatments. In five DMD patients treated for 28 days, grip strength increased on average by up to 4 kg (8.8 lb), suggesting a potential benefit. Dosing has now begun in a long-term follow-up study, LT-001, which plans to enroll patients who completed the Phase 1 trial, as well as new participants.

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No. 6 — FDA grants orphan drug status to GIVI-MPC for Becker MD

In January, the U.S. Food and Drug Administration (FDA) granted orphan drug status to IPS Heart‘s GIVI-MPC, a stem cell therapy being developed to treat Becker muscular dystrophy (BMD). The designation supports the development of rare disease drugs by providing the company with incentives, such as tax credits and market exclusivity. GIVI-MPC is a one-time treatment designed to create new muscle tissue with full-length dystrophin by reprogramming patient-derived stem cells using givinostat, a drug already approved for DMD under the brand name Duvyzat. Current gene therapies primarily deliver a smaller form of dystrophin, the company noted, but these treatments cannot regenerate muscle tissue or produce full-length dystrophin.

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No. 5 — Early preventive heart treatment extends survival in DMD

Preventive use of standard heart medications before cardiac problems arise significantly improves outcomes for males with DMD, according to data from the U.S.-based MD STARnet. Patients who received preventive heart treatment were less likely to develop left ventricular dysfunction (LVD), characterized by the heart’s left ventricle’s inability to pump blood effectively. These patients developed LVD later, and when it did occur, they lived longer than untreated patients. Despite these benefits, only about one-fourth of DMD patients received preventive heart medications. The survival and heart-protective benefits were consistent across care sites, medication types, and other clinical factors.

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No. 4 — LGMD gene-editing therapy shows promise in mice

Researchers have developed a gene-editing therapy targeting a common DYSF gene mutation linked to limb-girdle muscular dystrophy (LGMD) type 2B/R2. In patient-derived muscle stem cells, the treatment successfully corrected the genetic defect, restoring production of dysferlin, a protein required for muscle repair. In a mouse model harboring the same DYSF mutation, edited muscle stem cells transplanted into the muscles restored dysferlin levels and promoted muscle regeneration. Based on these promising results, the researchers are seeking funding to advance the approach into clinical trials.

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No. 3 — OPMD gene therapy BB-301 safely improves swallowing

In February, Benitec Biopharma announced that five people with oculopharyngeal muscular dystrophy (OPMD) had been treated with its experimental gene therapy, BB-301, in an ongoing clinical trial in New York City. Early data from the first two participants indicated the treatment was generally safe and improved swallowing function, a common issue in OPMD. BB-301 employs a “silence and replace” approach, inactivating the mutated PABPN1 gene while providing a healthy copy to patients’ cells. The study’s primary goals are to evaluate the safety of the treatment and its effects on swallowing. In a more recent update, BB-301 improved swallowing in the first three OPMD patients, regardless of their pretreatment swallowing difficulties. The trial is planned to enroll around 30 patients.

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No. 2 — Low-dose corticosteroids protect against muscle damage in LGMD, BMD

A U.S. pilot study found that once-weekly treatment with low doses of the corticosteroid prednisone improved muscle function and reduced markers of muscle damage and inflammation in people with LGMD and BMD. Blood analysis showed that prednisone shifted protein levels associated with muscle growth, repair, and immune regulation toward more normal patterns. Levels of creatine kinase, a marker of muscle damage, decreased, correlating with functional improvement, while lean mass remained stable or increased. Overall, weekly low-dose corticosteroid treatment was well tolerated.

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No. 1 — Elevidys gene therapy gives ‘hope’ to the DMD community

Nearly two years after its FDA approval, Sarepta Therapeutics‘ Elevidys (delandistrogene moxeparvovec-rokl), the first U.S.-authorized gene therapy for DMD, has shown strong clinical benefits and garnered a widespread positive reception. It’s fully approved for DMD children ages 4 and older who can walk. Data from Phase 3 EMBARK and other studies show that Elevidys slowed the decline in motor function and enabled better motor skills compared with untreated external controls. Follow-up data indicate slower functional loss and less MRI-detected muscle degeneration. Although the therapy was conditionally authorized for people with DMD, ages 4 and older, who cannot walk, the FDA removed this conditional approval in November and included new safety warnings and precautions in Elevidys’ prescribing label in light of two deaths related to the therapy reported last year.

Read more here.

At Muscular Dystrophy News Today, we hope our stories and reporting throughout 2025 positively impacted the lives of those with MD. We look forward to serving the community further in 2026.

A very happy New Year to all our readers!