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Patients with the rare disease typically present with hypertrophic cardiomyopathy and muscular hypotonia before the age of 1.

Findings are based on a case-control study carried out in Brazil.

The first phase 3 results from the CARTITUDE-4 trial of ciltacabtagene autoleucel (cilta-cel) vs standard of care in lenalidomide-refractory multiple myeloma (MM) demonstrated the former’s convincing superiority in several key outcomes.

James F. Howard Jr, MD, professor of neurology at the University of North Carolina at Chapel Hill, explains how nerve and muscle signaling in the neuromuscular junction go awry in myasthenia gravis.

Skin rashes are common among patients who complete hematopoietic cell transplantation (HCT).

Tycel Phillips, MD, hematologist-oncologist at City of Hope in Duarte, CA, discussed the utility of epcoritamab in diffuse large B-cell lymphoma (DLBCL) and the potential impacts the bispecific antibody's recent approval will have in the community oncology setting.

Investigators evaluated long-term outcomes among individuals who have eosinophilic esophagitis (EoE) and initiated first-line treatment with a food elimination diet after experiencing remission following stoppage of proton pump inhibitor monotherapy.

The autologous CAR T-cell therapy axicabtagene ciloleucel (axi-cel) demonstrated effectiveness and safety consistent with the ZUMA-5 trial when used in real-world settings to treat a broader population of patients with follicular lymphoma (FL).

Findings are based on a meta-analysis of 51 studies and 739 pregnancies.

Childhood myopia progression could be treated with low-dose atropine due to the safety and efficacy displayed in this study.

Research presented at the American Society of Clinical Oncology Annual Meeting found prostate-specific membrane antigen ligand positron emission tomography (PSMA-PET) to be associated with worse overall survival in prostate cancer without distant metastasis based on conventional imaging.

The Revolutionizing Atopic Dermatitis (RAD) meeting took place April 29 through May 1 in Washington, DC, with The American Journal of Managed Care® there to cover the meeting. Some of the top news you may have missed is right below.

Drs Derek van Amerongen, Soyini Hawkins, Roxie Cannon Wells, and Sharon Deans highlight triple-negative breast cancer among underrepresented patient populations.

The panel highlights initiatives to aid women in early cervical cancer diagnosis and intervention.

The phase 3 MATTERHORN trial examining roxadustat as an anemia treatment in patients with transfusion-dependent, lower-risk myelodysplastic syndrome did not meet its primary end point.

Treatment with the first-in-class direct and competitive telomerase inhibitor imetelstat resulted in statistically significant and clinically meaningful efficacy in patients with heavily transfusion dependent, non-del(5q) lower-risk myelodysplastic syndrome that is relapsed or refractory to erythropoiesis stimulating agents, according to findings from the phase 3 IMerge trial.

Researchers assessed overall survival outcomes in patients in France.

An expert panel navigates stigmas and barriers inhibiting patients from achieving optimal care for HIV.

Dr Adam C. Welch drives a discussion highlighting disparities in health care equity affecting patients who receive an HIV diagnosis.

New therapies could potentially target immune systems at play in certain types of diffuse large B-cell lymphoma.

Associations between intervention dosage and hospital use outcomes were observed among patients enrolled in a care management program serving individuals with complex needs.

Wanda Filer, MD, MBA, FAAFP, provides an overview of the potential outcomes for patients of all ages with RSV.

Key opinion leaders discuss ongoing clinical trials for respiratory syncytial virus vaccines.

The panel discusses potential therapy options for adults infected with respiratory syncytial virus.

The authors examined the association of diabetes with self-reported gaps in care coordination and self-reported preventable adverse events using data from a national sample of older adults.




















































