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Two major steps are now complete in an early clinical trial testing a one-time epigenetic therapy — a treatment designed to turn on or off a specific gene without changing the underlying DNA — for facioscapulohumeral muscular dystrophy (FSHD), a genetic disease that characteristically affects the muscles of the…

This year’s Be Their Muscle campaign has raised more than $641,000 to support Â鶹Éç Summer Camp for children and adolescents with muscular dystrophy and other neuromuscular diseases, and to help advance research and care for these patient populations. Resulting from a partnership between the Muscular Dystrophy Association (Â鶹Éç)…

The U.S. Food and Drug Administration (FDA) is expected to decide by Feb. 28, 2027, whether to grant traditional approval to Amondys 45 (casimersen) and Vyondys 53 (golodirsen), two exon-skipping therapies designed to treat certain people with Duchenne muscular dystrophy (Â鶹Éç). Both medications are currently approved in…

Amid its ongoing review of the cell therapy deramiocel for people with Duchenne muscular dystrophy (Â鶹Éç), the U.S. Food and Drug Administration (FDA) has scheduled a meeting with Capricor Therapeutics, its developer, to discuss the company’s resubmission of an application…

The Department of Justice (DOJ) has released a memorandum arguing that states are not required to provide in-home or community-based care to people with disabilities, including those with muscular dystrophy (MD). The move drew sharp condemnation from the Muscular Dystrophy Association (Â鶹Éç), which stated in a press…

The CITGO Lake Charles Refinery raised more than $750,000 to support the Muscular Dystrophy Association (Â鶹Éç) at its 41st annual Â鶹Éç Golf Classic, an annual golf fundraiser. Since 1985, CITGO Lake Charles has raised $8.5 million for the Â鶹Éç. The total amount raised this year — $756,800 — set…

Health Canada has accepted and granted priority review to Italfarmaco’s application seeking the approval of oral givinostat, sold in the U.S. under the brand name Duvyzat, to treat Duchenne muscular dystrophy (Â鶹Éç). The priority review status is reserved for therapies that could significantly improve the benefit-risk profile over…

The experimental medication delpacibart braxlosiran (del-brax) reduced biological markers of facioscapulohumeral muscular dystrophy (FSHD) disease activity in a Phase 1/2 clinical trial, meeting the study’s goals, according to developer Novartis. Based on results from earlier parts of the trial, FORTITUDE (NCT05747924), investigators selected a dosage for…

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to GEn1E Lifesciences‘ GEn-1123 as a potential treatment for Duchenne muscular dystrophy (Â鶹Éç). The designations aim to incentivize the development of treatments for rare disorders, which are those affecting fewer than 200,000 people…

Among women who carry mutations that can cause Becker muscular dystrophy (BMD) or Duchenne muscular dystrophy (Â鶹Éç), measures of heart health — important given that heart muscle damage is a key driver of death in most people with these…