Academia

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further innovations in safety, efficacy, and treatment strategies are needed.

New findings suggest that reducing the variant allele frequency of JAK2V617F should be a goal of treatment for polycythemia vera.

Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a favorable safety profile in young patients.

Patients with extensive-stage small cell lung cancer (SCLC) experienced clinically meaningful benefits with lurbinectedin plus atezolizumab vs atezolizumab in the phase 3 IMforte trial, according to findings being presented at the ASCO Annual Meeting.

Even a 2% decline in lung function predicted poor outcomes for patients with idiopathic pulmonary fibrosis (IPF) in a new study.

The tool could make it easier for clinicians to rule out melanoma without the need for excision.

The meta-analysis of 9 studies showed high sensitivity with slightly lower specificity with the less-invasive option for detecting lung complications in patients with the autoimmune disease.

The study, which used flow cytometry rather than molecular biology to identify circulating tumor cells (CTCs), found a significant positive correlation between the number of CTCs and the stage of disease.

Researchers hope that identifying the genomic alterations specific to transformed small cell lung cancer (SCLC) can help drive improvements in personalized treatments for these patients.

The analysis of 3 dozen studies found similarities and differences in motivating factors for and against clinical trial participation among patients with the 2 autoimmune diseases.

Increased levels of human leukocyte antigen G (HLA-G) were associated with inferior overall survival.

Many studies have examined how rheumatoid arthritis therapy affects the risk of metabolic comorbidities.

New research suggests classical biomarkers cannot discriminate between refractory and non-refractory rheumatoid arthritis.

This article appears in the April issue of Evidence-Based Oncology.

In a small study, lactotransferrin (LTF) was identified as a potentially important biomarker in extensive-stage small cell lung cancer (SCLC).

Several studies have been conducted, but questions remain about where such therapies will fit into multiple myeloma treatment strategies.

Inclusion of female and Hispanic/Latino patients has increased over time, but most trials of metabolic dysfunction-associated steatohepatitis (MASH) still take place in high-income countries and have majority-White patient populations.

Combining BET inhibitors and PARP inhibitors might help patients resistant to chemotherapy, the study suggests.

The new findings are the first to describe distributions and perturbations of low-density granulocyte and normal-density neutrophil subsets in myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), and idiopathic cytopenia of unknown significance.

The study found that just 20% of trials submitted to both the FDA and European Medicines Agency had matching evidence.

Investigators found that lipid metabolism disruptions spark T-cell dysfunction in patients with chronic lymphocytic leukemia.

Health care institutions with large numbers of Medicare and Medicaid patients may be constrained from offering cancer therapies made available under the accelerated approval pathway.

Regina Barragan-Carrillo, MD, a postdoctoral fellow at City of Hope Comprehensive Cancer Center, discussed findings that show 76% of renal cell carcinoma trials take place in wealthy countries, amid news that clinical trial access for the world's poor may become even more challenging.

Less than half of patients had undergone bone monitoring, suggesting a need for better clinical guidance and management of osteoporosis in adult men with Duchenne muscular dystrophy (DMD).

Patients with myelodysplastic syndromes (MDS) who undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT) are older and face worse outcomes compared with non-MDS patients, according to posters presented at the 2024 American Society of Hematology meeting.

The study demonstrated a poor prognosis overall for patients with acute myeloid leukemia (AML) who were previously treated with hypomethylating agents (HMAs) for myeloid neoplasms such as myelodysplastic syndromes.

Common measurements for modified adeno-associated viruses (AAVs), a gene therapy delivery method, showed substantial variation in accuracy and precision.

If the subcutaneous delivery method gains approval, an advantage daratumumab holds over isatuximab would be removed.