
Early tests show stem cells can be used to spark expression of a miniature version of the dystrophin protein.


“Personalized” Immunotherapy Appears Effective in Small Study of Patients With Metastatic Solid Tumors

Appendicular Lean Mass Index, Fat Mass Index Associated With Motor Function in DMD

Early tests show stem cells can be used to spark expression of a miniature version of the dystrophin protein.

Preexisting spinal damage and higher levels of inflammatory activity at baseline were linked with radiographic progression in axial spondyloarthritis treated with biologic disease-modifying antirheumatic drugs over 2 years regardless of treatment type.

High neutrophil to lymphocyte ratio (NLR) and platelet to lymphocyte ratio (PLR) are associated with worse progression-free survival and 5-year overall survival in patients with ovarian cancer.

Slowing the loss of ambulation in patients with Duchenne muscular dystrophy (DMD) may also mitigate worsening disease burden and overall function, according to a pair of posters presented at ISPOR 2024.

Combining the anti-CD38 monoclonal antibody with a standard backbone triplet should be the new standard of care for newly diagnosed patients not eligible for transplant, said lead investigator Thierry Facon, MD.

A recent review highlighted several candidate biomarkers for diagnosing axial spondyloarthritis associated with inflammatory bowel disease in early phases.

This study identifies limited engagement with equity among academic medical centers as they develop governance processes for artificial intelligence (AI)/machine learning and predictive technologies.

This editorial discusses positions for academic medical centers to consider when designing and implementing artificial intelligence (AI) tools.


Patients who took the weekly glucagon-like peptide 1 (GLP-1) receptor agonist were 21% less likely to experience major kidney disease events and 29% less likely to die due to cardiovascular causes.

Public hospitals were significantly more likely to sustain access to unprofitable services following 340B Drug Pricing Program participation, while nonprofit hospitals were mostly unaffected, according to a recent study.

The Inflation Reduction Act (IRA) has potential to shift incentives for drug manufacturers and impact the amount of long-term evidence generated for approved drugs, said Julie Patterson, PharmD, PhD, senior director of research at the National Pharmaceutical Council.

Stephanie T. Page, MD, PhD, UW Medicine Diabetes Institute, presented on ongoing research and growing interest in new male contraceptive options, such as an oral pill and a hormonal transdermal gel, at the American Urological Association 2024 Annual Meeting.

Survey results from more than 1500 patients with diabetes revealed that 57% believed glucagon-like peptide-1 (GLP-1) weight loss drugs alone could be a "silver bullet" for managing their health goals.

The novel intravesical drug delivery system releases erdafitinib locally within the bladder while limiting systemic toxicities.

Updated findings from SunRISe-1 also showed that all but 1 responder achieved complete response within 12 weeks of treatment with the targeted gemcitabine delivery system.

Speakers discussed the disparities in prostate-specific antigen (PSA) screening rates among different racial groups and explored the potential of free PSA percentage as a predictive marker for future prostate cancer risk.

A predictive model using plasma proteins as biomarkers predicted hepatocellular carcinoma development more accurately than a model based on traditional lifestyle risk factors in a recent study.

Adding olaparib to maintenance therapy with bevacizumab was associated with significantly longer survival for patients with ovarian cancer whose tumors have a BRCA-like genomic profile, but not among those with non-BRCA-like tumors, a study found.

"Off the shelf" CAR T-cell therapies could offer a solution in chronic lymphocytic leukemia, where T-cell exhaustion creates treatment challenges.

While the Dynamic International Prognostic Scoring System and bone marrow blasts may predict overall survival, the lack of certain mutations is also associated with a better prognosis for myeloproliferative neoplasm, unclassifiable (MPN-U).


Research indicated that worsened glycemic, blood pressure, and obesity control, as well as increased alcohol consumption, leveled lipid control, and persistent socioeconomic disparities may have contributed to the decelerated cardiovascular mortality decline in recent years.

A tumor board looked to studies in ovarian cancer for guidance on use of immunotherapy in a rare case of clear cell carcinoma of the cervix.

Patients who responded positively to shunt treatment experienced more beneficial changes in their heart's structure and function compared with nonresponders.