Clinical

Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disorder; a new report said that plasma lyso-sphingomyelin levels can be used to not only diagnose the rare disease but also predict severity and type.

Investigators reported comprehensive genomic features of patients with clear cell renal cell carcinoma, potentially giving providers a better understanding of the molecular features associated with the disease.

Dosing from pivotal phase 3 trials of tyrosine kinase inhibitors (TKIs) in chronic myeloid leukemia (CML) is not optimal for real-world patients who experience different efficacy and adverse events (AEs).

Maralixibat is the first treatment approved for cholestatic pruritus in patients with Alagille syndrome who are 1 year or older.

A new study suggests the lower-cost, more patient-friendly assessment method may be a useful tool for clinicians.

Therapies that block adenosine triphosphate (ATP) can reduce the irritation that causes cough, and some currently being studied also don’t have taste side effects that can confound clinical trial results, explained Ron Eccles, BSc, PhD, DSc, emeritus profess at Cardiff University.

Researchers identified the frequency with which acute medication overuse occurs among migraineurs and factors associated with the condition.

This new study adds to the literature by giving the first long-term data in these populations.

A look at the existing literature suggests more questions than answers remain when it comes to the potential use of the cholinergic anti-inflammatory pathway in connective tissue diseases (CTDs).

With the Oncology Care Model (OCM) slated to end in 2022, the successor model is still not in place. According to panelists, there is likely going to be a gap after OCM ends and the new model begins, but more importantly, practices should prepare for the new model to be mandatory.

Study results released earlier this month showcased results in children and adults with uncontrolled asthma. The findings were presented at the European Respiratory Society (ERS) 2021 International Congress.

In the opening sessions at the Patient-Centered Oncology Care® conference, held September 23-24 both virtually and in Nashville, Tennessee, speakers focused on addressing disparities by getting involved and engaged with the community.

The reports suggest patients with rheumatoid arthritis (RA) could experience secondary neurological adverse events, though the side effect appears to be rare.

Intravenous belimumab had benefits, but also resulted in more health care utilization due to the nature of its administration.

A team of Australian researchers created and validated a model that may boost efficiency of type 1 diabetes (T1D) progression screening.

In a recent paper, researchers outlined the utility of minimal residual disease (MRD) among patient with acute lymphoblastic leukemia (ALL) and how its monitoring at specific timepoints has proven useful for stratifying patients and helping to ensure appropriate treatment decisionsMRD

Two posters presented at the European Society of Medical Oncology highlighted the unmet needs of cancer survivors.

Ruxolitinib cream, to be sold under the name Opzelura, is the first topical Janus kinase inhibitor cream for the treatment of atopic dermatitis.

Chronic graft-vs-host disease (GVHD) is a condition that can occur after an allogeneic stem cell transplant in which the donated cells initiate an immune response and attack the transplant recipient’s organs.

Among patients with COVID-19, chronic obstructive pulmonary disease and asthma were more prevalent than allergic rhinitis.

New data from the CREDENCE trial shows that the sodium glucose co-transporter 2 (SGLT2) inhibitor canagliflozin may reduce hyperkalemia risk in people with type 2 diabetes and chronic kidney disease who are using RAAS inhibitors.

Sanofi and Regeneron had previously announced an overall survival advantage of 29% over chemotherapy alone; the combination also had a progression-free survival benefit of 46%.

A review of current data identified areas of progress in Bruton tyrosine kinase (BTK) inhibition for patients with chronic lymphocytic leukemia (CLL).

Unlike the success seen in other cancer types, biomarker-driven treatment advances in gastroesophageal adenocarcinoma (GEA) have been hindered by its genomic heterogeneity. A significant proportion of patients also develop resistant subclones through selective pressure, resulting in disease progression.

Patients who are treated with direct-acting antivirals to treat hepatitis C virus (HCV) are still at risk of developing hepatocellular carcinoma (HCC).