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The phase 3 PEGASUS trial found that patients with paroxysmal nocturnal hemoglobinuria (PNH) may still experience anemia following recommended therapies, underscoring the need for further research.

Mark Bleackley, PhD, chief scientific officer of Incannex, discusses in further detail the need for a pharmacotherapy like IHL-42X in obstructive sleep apnea (OSA) and the medication's underlying mechanisms.

A new report offers safety and efficacy data on a population with generalized myasthenia gravis (gMG) not represented in the drug’s pivotal phase 3 trial.

A cross-sectional study found that static pain measures could be useful for predicting fatigue severity in patients with sickle cell disease (SCD).

Researchers of a new study explore what areas of chronic lymphocytic leukemia (CLL) treatment and care are important to patients and identify key areas for improvement.

For this analysis, investigators were interested in patient non–small cell lung cancer (NSCLC)–related outcomes from antibiotic administration before immunotherapy alone or immunotherapy and chemotherapy.

Liz Kwo, MD, MBA, MPH, chief commercial officer, Everly Health, and faculty lecturer, Harvard Medical School, discusses ways that wearable devices and health apps are improving value-based care.

A new report provided updated figures estimated the birth prevalence of spinal muscular atrophy (SMA) in the US.

A literature review plus in-person visits helped identify barriers to quality rheumatoid arthritis care, as well as effective interventions to improve care quality.

Almost half of all Americans struggle to afford quality health care and prescription medications; the cyberattack on UnitedHealth Group's Change Healthcare unit will cost the company between $2.3 billion and $2.45 billion in 2024; CMS releases the final part 2 guidance for plan outreach and education for the Medicare Prescription Payment Plan.

Investigators hoped to use large-scale proteomics to help predict hypertensive disorders of pregnancy (HDP), using blood proteins obtained from individuals in their first trimester of pregnancy—but success has been elusive.

In this interview from our coverage of the European Hematology Association 2024 Congress, we spoke with Brian Koffman, MDCM, DCFP, FCFP, DABFP, MSEd, executive vice president and chief medical officer of the CLL Society and himself a survivor of chronic lymphocytic leukemia (CLL), to discuss treatment advancements and the importance of addressing both unmet needs and patient treatment preferences.

Ron Grunstein, MD, PhD, head of sleep and circadian research, Woolcock Institute of Medical Research, details results from the ALKS 2680 trial and the study's next steps.

Against the backdrop of a case report, pembrolizumab was investigated as immunotherapy for undifferentiated pleomorphic soft tissue sarcoma.

A new case report details the successful use of efgartigimod as a rescue medication in a patient with therapy-refractory myasthenic crisis.

A trio of experts discuss the challenges of diagnosing and managing paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening disorder.

We recently spoke with Tycel Phillips, MD, associate professor, Division of Lymphoma, Department of Hematology & Hematopoietic Cell Transplantation, City of Hope, about his team’s interim analysis of their dose-escalation study of glofitamab against relapsed/refractory B-cell non-Hodgkin lymphoma.

A retrospective database analysis demonstrated the high clinical burden experienced by patients with severe deficiencies of ADAMTS13 and thrombotic thrombocytopenic purpura (TTP)–related diagnoses.

Up to one-third of Americans have been affected by data stolen through the Change Healthcare cyberattack; research highlights potential cognitive and psychiatric benefits of semaglutide; over 40 Iowa hospitals have ended their labor and delivery services.

Kelly Harris, APRN, an advanced practice registered nurse at Cincinnati Children's Hospital Medical Center, discusses using mobile care units to deliver dermatology care and education to underserved communities and how health systems can implement them.

In this final segment of our interview with Ontada's Jessica K. Paulus, ScD, she explains how her team's data presented at the American Society of Clinical Oncology annual conference may translate to the real world.

Learn more about the 2025 Physician Fee Schedule, new health equity initiatives, improving maternal health, and pharmaceutical innovations at the Center on Health Equity & Access.

Results from a national survey of patients with Duchenne muscular dystrophy (DMD) and their families found that patients and families want to be informed early about endocrine complications associated with glucocorticoid treatment.

The aflibercept 8 mg injection improves patient care by allowing longer intervals between treatments, explains Jose A. Martinez, MD, Austin Retina Associates.

Dual Therapy Well-Tolerated but Presents Little Benefit to Infantile-Onset SMA
A multicenter pilot study concluded that dual therapy in spinal muscular atrophy (SMA) does not provide further benefits to infants at risk for SMA type 1.












