CHARLESTOWN, Mass. — December 16, 2025 — Leads & Copy — Solid Biosciences Inc. (Nasdaq: SLDB) announced today that the U.S. Department of Health and Human Services (HHS) has officially added Duchenne muscular dystrophy (Duchenne) to the Recommended Uniform Screening Panel (RUSP), which is a list of conditions recommended for universal newborn screening across the United States.
Solid Biosciences has been a supporter of Parent Project Muscular Dystrophy’s (PPMD) committee for nearly a decade, which is dedicated to implementing newborn screening for Duchenne. This began with a state-level pilot program in New York that was completed in 2021. The addition of Duchenne to the RUSP is expected to accelerate detection efforts, potentially improving outcomes for those living with Duchenne through earlier access to specialists, supportive intervention, and treatment options.
Annie Ganot, SVP of Patient Advocacy and Co-founder of Solid Biosciences, stated that Solid is honored to stand alongside PPMD in championing the inclusion of Duchenne on the RUSP. Ganot noted that this milestone is the result of years of evidence generation, advocacy, and commitment from families, clinicians, researchers, and industry partners. She added that this achievement marks a transformative moment for newborn screening, ensuring earlier diagnosis and access to vital resources for newly diagnosed families. Solid Biosciences remains focused on delivering its investigational gene therapy, SGT-003, to the Duchenne community.
Duchenne is a genetic muscle-wasting disease predominantly affecting boys, with symptoms usually appearing between three and five years of age. It is a progressive, irreversible, and ultimately fatal disease that affects approximately one in every 3,500 to 5,000 live male births and has an estimated prevalence of 5,000 to 15,000 cases in the United States alone.
SGT-003 is an investigational gene therapy containing a differentiated microdystrophin construct and a next-generation capsid, AAV-SLB101, designed to target integrin receptors. Nonclinical studies have demonstrated enhanced cardiac and skeletal muscle transduction with decreased liver targeting. SGT-003’s microdystrophin construct includes the R16/17 binding domain, which localizes nNOS to the muscle membrane. Nonclinical studies have indicated that nNOS can improve blood flow to the muscle, thereby reducing muscle breakdown from ischemia and muscle fatigue. These design features suggest that SGT-003 could be a potential best-in-class investigational gene therapy for the treatment of Duchenne.
Solid Biosciences is focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne, SGT-212 for Friedreich’s ataxia (FA), SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT), and SGT-601 for TNNT2-mediated dilated cardiomyopathy. The company is also developing innovative libraries of genetic regulators and enabling technologies. Solid Biosciences’ mission is to improve the daily lives of patients living with devastating rare diseases.
Solid Biosciences Investor Contact:
Nicole Anderson
Director, Investor Relations and Corporate Communications
investors@solidbio.com
Media Contact:
Glenn Silver
FINN Partners
glenn.silver@finnpartners.com
Source: Solid Biosciences
