Solid Biosciences Announces Duchenne Muscular Dystrophy Added to National Recommended Uniform Screening Panel by the U.S. Department of Health and Human Services
Rhea-AI Summary
Solid Biosciences (Nasdaq: SLDB) announced that Duchenne muscular dystrophy was added to the U.S. Recommended Uniform Screening Panel (RUSP) on Dec. 16, 2025, making it recommended for universal newborn screening.
Solid noted its long-term advocacy role, including participation on PPMD’s steering committee and a New York state pilot completed in 2021. The company said RUSP inclusion is expected to accelerate detection and enable earlier access to specialists, supportive interventions, and treatments.
Solid reiterated its focus on advancing its investigational gene therapy SGT-003 for Duchenne while celebrating earlier diagnosis and resource access for families.
Positive
- Duchenne added to RUSP on Dec. 16, 2025
- Solid served on PPMD steering committee and led NY pilot completed 2021
- RUSP inclusion expected to accelerate earlier detection and specialist access
Negative
- Nationwide implementation timing for newborn screening is not specified
News Market Reaction 2 Alerts
On the day this news was published, SLDB gained 1.93%, reflecting a mild positive market reaction. Argus tracked a trough of -3.4% from its starting point during tracking. Our momentum scanner triggered 2 alerts that day, indicating moderate trading interest and price volatility. This price movement added approximately $9M to the company's valuation, bringing the market cap to $453M at that time.
Data tracked by StockTitan Argus on the day of publication.
Key Figures
Market Reality Check
Peers on Argus
Pre-news moves in close biotech peers were mixed, with AURA (-1.59%), FULC (-1.81%) and LXRX (-1.64%) down, while DMAC (+1.62%) and ALMS (+2.15%) were up, indicating no clear sector-wide direction tied to this announcement.
Historical Context
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| Dec 08 | Scientific conference | Positive | -1.0% | New data and presentations on AAV-SLB101 and cardiac gene therapy pipeline. |
| Dec 01 | Equity compensation | Neutral | -2.1% | Inducement RSU grants to new hires under Nasdaq Listing Rule 5635(c)(4). |
| Dec 01 | Regulatory designation | Positive | -4.0% | FDA Rare Pediatric Disease designation for SGT-212 gene therapy for FA. |
| Nov 17 | Licensing deal | Positive | +2.4% | Non-exclusive global license for AAV-SLB101 with supportive safety data. |
| Nov 11 | Investor conferences | Neutral | +9.2% | Participation in Jefferies and Piper Sandler healthcare investor conferences. |
Recent SLDB news has often been followed by modest and sometimes negative price moves, even after seemingly positive clinical or partnership updates.
Over the past month, Solid Biosciences has reported several pipeline and corporate developments. On Nov 17, 2025, it signed a non‑exclusive AAV‑SLB101 licensing deal with Andelyn, with shares rising 2.36%. Earlier, an Nov 3, 2025 8‑K detailed positive interim SGT‑003 Duchenne data. The company also obtained FDA Rare Pediatric Disease designation for SGT‑212 on Dec 1, 2025, yet the stock fell 4.05%. Today’s RUSP inclusion for Duchenne fits this pattern of significant ecosystem progress around its gene therapy focus.
Market Pulse Summary
This announcement highlights a policy milestone as Duchenne muscular dystrophy joins the U.S. Recommended Uniform Screening Panel, potentially enabling earlier diagnosis and access to care. For Solid, it reinforces strategic focus on Duchenne and its investigational gene therapy SGT‑003, following recent positive interim data disclosed in regulatory filings. Investors may track how broader newborn screening implementation, ongoing clinical progress, cash runway to the first half of 2027, and any future regulatory updates intersect with this evolving landscape.
Key Terms
recommended uniform screening panel regulatory
newborn screening medical
gene therapy medical
AI-generated analysis. Not financial advice.
CHARLESTOWN, Mass., Dec. 16, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today shared that the U.S. Department of Health and Human Services (HHS) officially added Duchenne muscular dystrophy (Duchenne) to the Recommended Uniform Screening Panel (RUSP), the list of conditions recommended for universal newborn screening across the United States.
For nearly a decade, Solid has been a staunch supporter and a steering committee member of Parent Project Muscular Dystrophy’s (PPMD) committee dedicated to implementing newborn screening for Duchenne, beginning with a state-level pilot program in New York that was completed in 2021. Adding Duchenne to the RUSP is expected to accelerate detection efforts, leading to earlier access to specialists and supportive intervention and treatment options, potentially improving outcomes for those living with Duchenne.
“At Solid, we are honored to stand alongside PPMD in championing the inclusion of Duchenne on the RUSP, a milestone born from years of rigorous evidence generation, tireless advocacy, and the unwavering commitment of families, clinicians, researchers, and industry partners,” said Annie Ganot, SVP of Patient Advocacy and Co-founder of Solid Biosciences. “This achievement marks a transformative moment for newborn screening, ensuring earlier diagnosis and access to vital resources for newly diagnosed families. As we celebrate this progress, we remain deeply focused on delivering our differentiated, investigational gene therapy, SGT-003, to the Duchenne community with urgency and care, driven by the belief that continued innovation in the treatment landscape can bring new hope to families everywhere.”
About Duchenne
Duchenne is a genetic muscle-wasting disease predominantly affecting boys, with symptoms usually appearing between three and five years of age. Duchenne 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.
About SGT-003
SGT-003 is an investigational gene therapy containing a differentiated microdystrophin construct and a proprietary, next-generation capsid, AAV-SLB101, which was rationally designed to target integrin receptors, and has shown enhanced cardiac and skeletal muscle transduction with decreased liver targeting in nonclinical studies. SGT-003’s microdystrophin construct uniquely includes the R16/17 binding domain, which localizes nNOS to the muscle membrane. Nonclinical studies have shown that nNOS can improve blood flow to the muscle thereby reducing muscle breakdown from ischemia and muscle fatigue. Together, these design features suggest that SGT-003 could be a potential best-in-class investigational gene therapy for the treatment of Duchenne.
About Solid Biosciences
Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne muscular dystrophy (Duchenne), SGT-212 for Friedreich’s ataxia (FA), SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT), SGT-601 for TNNT2-mediated dilated cardiomyopathy and additional fatal, genetic cardiac diseases. The Company is also focused on developing innovative libraries of genetic regulators and other enabling technologies with promising potential to significantly impact gene therapy delivery cross-industry. Solid is advancing its diverse pipeline and delivery platform in the pursuit of uniting experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted by Duchenne, Solid’s mission is to improve the daily lives of patients living with devastating rare diseases. For more information, please visit www.solidbio.com.
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding future expectations, plans and prospects for the Company; the ability to successfully achieve and execute on the company’s goals, priorities and achieve key clinical milestones; the Company’s programs for neuromuscular and cardiac diseases, including its SGT-003 candidate, its pipeline of capsid products, including AAV-SLB101, and other clinical and pre-clinical programs and expectations for clinical development, initiation and enrollment in clinical trials, dosing, availability of clinical trial data and potential accelerated approval; and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” “working” and similar expressions. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the company’s ability to advance SGT-003 and its other clinical and preclinical programs and advance AAV-SLB101 and its capsid libraries on the timelines expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; replicate in clinical trials positive results found in preclinical studies and early-stage clinical trials of the company’s product candidates; obtain, maintain or protect intellectual property rights related to its capsid libraries and product candidates; compete successfully with other companies that are seeking to develop capsids, capsid libraries, Duchenne, Friedreich’s ataxia and other neuromuscular and cardiac treatments and gene therapies; achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the company’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in the company’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company’s views as of the date hereof and should not be relied upon as representing the company’s views as of any date subsequent to the date hereof. The company anticipates that subsequent events and developments will cause the company's views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so.
Solid Biosciences Investor Contact:
Nicole Anderson
Director, Investor Relations and Corporate Communications
Solid Biosciences Inc.
investors@solidbio.com
Media Contact:
Glenn Silver
FINN Partners
glenn.silver@finnpartners.com
FAQ
What does the RUSP addition mean for Duchenne and newborns as of Dec. 16, 2025 for SLDB?
How has Solid Biosciences (SLDB) been involved in newborn screening for Duchenne?
Will RUSP inclusion change access to treatments for Duchenne patients according to SLDB?
How does this announcement affect Solid Biosciences' development of SGT-003 (SLDB)?
Does the announcement provide a timeline for nationwide newborn screening implementation after RUSP addition?
