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Victor Ambros and Gary Ruvkun received the 2024 Nobel Prize in Physiology or Medicine for discovering microRNA and its role in gene regulation.

Survodutide is a glucagon/glucagon-like peptide-1 (GLP-1) receptor dual agonist that activates the glucagon and GLP-1 receptors to better control metabolic function.

Women were also more likely to report concerns of high wait times prior to receiving care.

At the 2024 World Conference on Lung Cancer, Benjamin Besse, MD, PhD, discussed the findings of the phase 3 CARMEN-LCO3 trial, which led to the discontinuation of the study drug by the manufacturer.

Setrusumab could offer a rapid and clinically meaningful decrease in fracture rate in individuals with osteogenesis imperfecta.

Treosulfan showed superior benefits to busulfan as a conditioning regimen before allogenic hematopoietic stem cell transplantation (allo-HSCT).

Zodasiran is an RNA interference therapy targeting the ANGPTL3 loss-of-function mutation.

Though both treatments come with positives and negatives, ultimately, individual patient clinical presentation and accessibility will determine the proper treatment for myasthenic crisis.

Eligible patients can receive a 30-day supply of covered drugs and medical supplies that can be renewed every 30 days for as long as an Emergency Prescription Assistance Program (EPAP) is active.

Research showed that children born via assisted reproductive technology were 36% more likely to develop heart defects.

Major biomarkers for cognitive impairment were observed in hospitalized patients with neurological symptoms.

Currently, standard first-line chemotherapy with or without immunotherapy for patients with non-small-cell lung cancer (NSCLC) with HER2 mutations has limited efficacy.

Fedratinib is an orally available, small molecule inhibitor of JAK-2 approved for treatment of myelofibrosis.

By utilizing a lower dose, patients can avoid potential adverse events and high financial burden.

Eculizumab (Soliris; Alexion), a C5 inhibitor preventing cleavage into C5a and C5b, is widely considered the first-line therapy for high-risk TA-TMA.

Currently, Danon disease has a poor prognosis and does not have any pharmaceutical therapeutics for treatment or management.

Pharmacists and health care providers can counsel around vitamin D supplements and natural intake to increase a child’s vitamin D consumption.

The findings may offer patients treatment options beyond symptom palliation.

The weight loss drug continues to show promise in treating a variety of other conditions.

RSV vaccination could be cost-effective among adults 60 years and older, with the ability to decrease vaccine costs and sustain efficacy.

Navenibart was developed to aid treatment of hereditary angioedema (HAE) to provide swift and sustained HAE attack prevention through administration every 3 to 6 months.

Treatment for primary coenzyme Q10 (CoQ10) deficiency can include high-dose oral CoQ10 supplementation, but not all patients respond to this treatment.

Zanubrutinib demonstrated sustained responses in patients with relapsed/refractory (R/R) chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL).

The results validate the protective benefits of the sodium–glucose cotransporter-2 inhibitors on renal function for those with type 2 diabetes.

The accelerated course was safe and effective and did not increase complications in patients with breast cancer post-mastectomy undergoing breast reconstruction.

Approximately 75% of adults trust their physicians, nurses, and pharmacists either a great deal or a lot, highlighting the importance of pharmacists for patient education.

MDL-101 is a proposed novel precision medicine that targets the LAMA1 gene, causing LAMA2 congenital muscular dystrophy type 1a.

The guidelines mark a shift in practice by recommending a multimodal approach to opioid prescribing practices.