The gene therapy RGX-202 at lower dose was well tolerated by three boys with Duchenne muscular dystrophy (DMD) in an early clinical trial, and biomarker data suggest the treatment is working as intended. Regenxbio, the company developing RGX-202, expects to administer a higher dose of…
News
Researchers have created a three-dimensional (3D) artificial muscle model, derived from patient cells, that accurately replicates the damage observed in people with Duchenne muscular dystrophy (DMD). This new model, the scientists believe, will enable more efficient testing of treatments that could potentially reverse such muscle damage. The findings were…
Capricor Therapeutics intends to ask the U.S. Food and Drug Administration (FDA) to approve its experimental cell therapy CAP-1002 for treating Duchenne muscular dystrophy (DMD) in 2025. The company recently completed a meeting with the agency, which confirmed that data from the ongoing Phase 3 clinical…
A Phase 2 clinical trial testing the investigational oral medication EDG-5506 in people with Becker muscular dystrophy (BMD) is expanding, with plans to enroll 120 additional adults with the genetic disorder. According to EDG-5506’s developer Edgewise Therapeutics, enrollment in the expanded study is ongoing. The company is hosting…
AMO-02 (tideglusib), an experimental oral therapy for certain muscular dystrophies, showed promise in boosting motor and cardiac muscle health in a mouse model of Duchenne muscular dystrophy (DMD). According to AMO Pharma, the treatment’s developer, AMO-02 also improved the animals’ metabolism and cognitive abilities. “These studies…
Covering topics such as mobility equipment and the role of physical therapy, a new, free, online CureDuchenne course seeks to provide guidance and support to Duchenne muscular dystrophy (DMD) caregivers. Specifically, the virtual CureDuchenne Caregiver Course seeks to help caregivers of those newly diagnosed with DMD —…
The conditional approval of Translarna (ataluren) in Europe as a treatment for Duchenne muscular dystrophy (DMD) — in place for nearly a decade — should now not be renewed, a committee of the European Medicines Agency (EMA) has recommended. The EMA’s Committee for Medicinal Products for Human Use…
Fulcrum Therapeutics has completed patient enrollment for its Phase 3 clinical trial evaluating losmapimod as a potential treatment for people with Facioscapulohumeral muscular dystrophy (FSHD). The study, dubbed REACH (NCT05397470), is evaluating the efficacy and safety of losmapimod against a placebo in FSHD adult patients across…
Entrada Therapeutics has awarded $25,000 grants to three U.S. nonprofits to aid in their efforts to combat healthcare disparities in the Duchenne muscular dystrophy (DMD) community. The goal of these nonprofits, as inaugural recipients of the biopharmaceutical company’s DREAMS — Diversity, Representation, Equity and Advocacy Matters — grant…
The European Medicines Agency (EMA) has started reviewing an application to market givinostat for Duchenne muscular dystrophy (DMD). “Now that our market authorization application is under review, we are looking forward to working with the regulatory authorities to bring this treatment option to patients,” Paolo Bettica, MD, PhD,…
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