Providers

The recent availability of 3 FDA-approved treatments for patients with spinal muscular atrophy (SMA) has dramatically altered patient outcomes and provided a wealth of new avenues for SMA research.

In a single-center, retrospective analysis, echocardiography demonstrated low sensitivity but high specificity for diagnosing pulmonary hypertension (PH) in advanced chronic obstructive pulmonary disease (COPD).

Patients with diverse skin types can experience diagnostic delays, access to care issues, and quality-of-life burdens that a navigator program may be able to help alleviate.

To stress the importance of considering pulmonary complications in sickle cell disease (SCD), researchers compiled an overview on the state of research and available treatment options for patients with SCD associated with pulmonary hypertension.

The study also identified similar 12-month outcomes between torsemide and furosemide regardless of heart failure (HF) type.

Alexander Fay, MD, PhD, joined for a Q&A about the current state of spinal muscular atrophy (SMA) treatment and the unanswered questions in the field.

In these phase 3 findings presented at ACR 2023, the safety and efficacy of this drug was proven in American College of Rheumatology-20 (ACR20), ACR50, and Disease Activity Score in 28 joints with Erythrocyte Sedimentation Rate responses.

A study from investigators in Italy sheds further light on the relationship between physical activity and diabetic polyneuropathy risk in patients with type 1 diabetes.

The use of base editing to generate universal, off-the-shelf CAR T cells is a promising approach for relapsed leukemia, with potential implications for the future of gene therapy.

A recent review aimed to characterize the relationship between tumor burden and clinical outcomes in patients treated with chimeric antigen receptor (CAR) T-cell therapy, highlighting the potential mechanisms of high tumor burden impacting CAR T-cell failure.

A retrospective, exploratory analysis identifies biomarkers predictive of spinal muscular atrophy (SMA) response to nusinersen and suggests the efficacy of using machine learning algorithms to anticipate patient outcomes.

In a recent study, reducing dietary sodium demonstrated a blood pressure–reducing effect similar to that of a commonly used first-line antihypertensive medication.

Alyson Moadel-Robblee, PhD, deputy director of community engagement and cancer health equity at Montefiore Einstein Comprehensive Cancer Center, discussed the center's evolving patient distress screening process and the Bronx Oncology Living Daily support program for patients with cancer.

A Biologics License Application has been submitted to the FDA review for sotatercept in the treatment of pulmonary arterial hypertension.

The study findings provide the first in-depth look at the relationship between chromosomal changes in tumor cells and immune components of the tumor microenvironment.

The FDA has approved pembrolizumab (Keytruda) in combination with fluoropyrimidine- and platinum-containing chemotherapy for the first-line treatment of adult patients with locally advanced unresectable or metastatic, HER2-negative gastric or gastroesophageal junction adenocarcinoma.

The tool assesses the risk of heart attack, stroke, and, for the first time, heart failure, offering a more comprehensive perspective on cardiovascular health.

Michael Yim, MD, board-certified family physician, discussed the need for and logistics of collaboration between oncologists and primary care providers.

According to researchers, the most prevalent risk factors in children with type 1 spinal muscular atrophy (SMA) are hypercalciuria and elevated urine specific gravity.

Investigators note that prospective research is needed to confirm the efficacy of vitamin D supplements in preventing chemotherapy-induced peripheral neuropathy in patients with breast cancer.

Michael Fang, PhD, researcher and assistant professor in the division of Cardiovascular and Clinical Epidemiology at Johns Hopkins University, discussed recent findings in the type 1 diabetes (T1D) space that may alter the way providers address diabetes diagnoses.

New research highlighted how aspirin is unnecessary in antithrombotic regimens for patients with advanced heart failure and fully magnetically levitated left ventricular assist devices (LVADs).

An artificial intelligence (AI)–based prediction model correctly predicted outcomes for 78% of patients with high-grade serous ovarian carcinoma, with an accuracy of 80%.

A retrospective analysis of data from the US National Inpatient Sample revealed the prevalence of fibromyalgia and chronic fatigue syndrome was significantly increased in patients with IBS compared with those without IBS.

These new findings are important as α-blockers (ABs) are the most prescribed class of medications for patients with benign prostatic hyperplasia (BPH), but the cardiovascular safety profile of this treatment is not well understood for this patient population.