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Enthusiasm abounded at the recent European Hematology Association 2024 Congress about whether European regulators will begin to consider minimal residual disease (MRD) as an end point.

Accelerated approval was originally granted in June 2023 for patients aged 4 to 5 years, indicating there was an unmet clinical need for a potentially life-saving treatment for the rare genetic muscle disorder.

Sickle cell trait (SCT) and pregnancy each exacerbate the symptoms of COVID-19. As a result, pregnant individuals with SCT may have a higher risk of COVID-19 infection.

Atopic dermatitis significantly impairs female patients' sexual function, reproductive desires, and overall quality of life.

This study highlights the lack of clear guidelines for breast cancer management in transgender men after chest contouring surgery. While data is limited, the authors suggest adapting existing recommendations for cisgender women and implementing risk management strategies like pre-surgery evaluation and education.

Taking falsified semaglutide (Ozempic; Novo Nordisk) can lead to ineffective treatment due to incorrect dosing, contamination, and the use of unknown ingredients, the World Health Organization (WHO) is warning.

The phase 3 GENEr8-1 study demonstrated that valoctocogene roxaparvovec, an AAV5-vectored gene therapy, effectively enabled endogenous FVIII production in adults with severe hemophilia A without developing clinically meaningful FVIII inhibitors.

The US Preventive Services Task Force recommends that kids 6 years and older with a high body mass index receive intensive behavioral interventions; Amazon’s pharmacy unit is expanding eligibility for RxPass to patients enrolled in Medicare insurance plans; Sen Bernie Sanders (I, Vermont) is trying to lower the prices of popular antiobesity drugs.

A poster presented at the 2024 American Society of Clinical Oncology annual meeting argued the benefits that Medicaid expansion can bring to patients with melanoma.

Patients with stage IV epithelial ovarian cancer (OC) treated with surgery and chemotherapy exhibit the best prognosis.

People receiving hypomethylating agent (HMA) therapy spent 33 more days at home than people receiving anthracycline-based therapy in the first year after diagnosis.

Pfizer’s investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, did not meet its primary end point of improvement in motor function in ambulatory patients with Duchenne muscular dystrophy (DMD).

A prospective cohort study, using data from the UK Biobank, suggested that the Mediterranean diet could provide benefits to patients with multiple sclerosis.

Older adults who had colorectal surgery were found to have reduced postoperative complications if they started the Geriatric Oncology Service.

The rate of uninsured Americans will rise to 8.9% over the next decade; Senator Bernie Sanders (I, Vermont) wants a government watchdog to investigate why women are still being charged for contraception considered free under federal law, and proposed Medicare Advantage Star Ratings target top performers.

A new prevalence study shows more people are living beyond age 65 with type 1 diabetes (T1D).

Determining the roles that various inflammatory markers and pathways play in lower-risk myelodysplastic syndromes (MDS) could lead to therapies that keep disease progression at bay.

A panel of ophthalmologists defined points of consensus for suprachoroidal space injections based on current published evidence.

In a European expert consensus statement, researchers discover the exciting breakthroughs for patients with alopecia areata (AA). This article explores the unlocked potential of combination therapies and targeted biologics to regrow hair and transform lives.

This study provides a comprehensive review of both established and emerging prognostic markers in chronic lymphocytic leukemia (CLL), and their role in predicting disease, treatment response, and overall survival.

June 19 is marked as World Sickle Cell Awareness Day, granting the opportunity to reflect on the current state of treatment, access, equity, and more that impacts patients living with sickle cell disease.

Study findings show eculizumab is effective and well tolerated in reducing disease burden in adolescents with refractory anti-acetylcholine receptor antibody–positive generalized myasthenia gravis.

The home-based care model offers a promising strategy for improving quality of care, while also reducing health care spending.

Encouraging trends were seen with osimertinib and platinum-pemetrexed chemotherapy in patients with EGFR-mutated advanced non–small cell lung cancer (NSCLC) in the phase 3 FLAURA2 study.

The albumin-bilirubin score is a reliable predictor of treatment response for hepatocellular carcinoma (HCC) after yttrium 90 transarterial radioembolization (TARE).

A poster from the 2024 Revolutionizing Alopecia Areata, Vitiligo, and Eczema (RAVE) conference held in Chicago, Illinois presented results of a clinical trial for patients with alopecia areata (AA). The trial used rilecitinib and showed promising results in patients with AA after most patients achieved significant hair regrowth following 15 months of treatment.

The treatment regimen not only significantly improved progression-free survival (PFS) for patients with advanced stage classic Hodgkin lymphoma but was also better tolerated compared with a 20-year-old regimen being used.

Vaccination against seasonal influenza and respiratory syncytial virus (RSV) in patients with high-risk heart failure was found safe with low incidence of mild adverse events (AEs).

A Chinese study shows hypertension management led by nonphysicians can be safe and effective.

Over $3 billion in payments were issued to health care providers and suppliers; a recent poll highlights growing demand to alleviate medical debt; preliminary data show a decrease in uninsured individuals since 2019.