Luke Halpern, Associate Editor

Steven Pipe, MD, explains the long-term efficacy data surrounding etranacogene dezaparvovec in patients with hemophilia B.

Amlenetug could provide a treatment option for patients with multiple system atrophy, a progressive and rare condition that causes damage to the brain’s nerve cells.

A one-time infusion of etranacogene dezaparvovec-drlb led to sustained bleeding control and a reduction in factor IX prophylaxis in patients with hemophilia B.

PP-01 could become a first-in-class treatment for patients with cannabis use disorder.

Therapies like the newly approved suzetrigine, a nonopioid NaV1.8 pain signal inhibitor, could revolutionize pain management and mitigate the risks associated with opioid use.

The agency will now review the novel PCSK9 inhibitor for possible approval in patients with or at high risk of atherosclerotic cardiovascular disease.

Building on a previously granted orphan drug designation, the latest FDA action for amezalpat puts the drug in a position for regulatory approval.

Cytopenic myelofibrosis is characterized by poorer prognosis and more severe anemia and thrombocytopenia compared with proliferative myelofibrosis.

The approval is based on positive clinical trial results that indicated deep and durable reductions in plexiform neurofibroma.

Emerging data indicate that COVID-19 is also characterized by inflammation present in the heart and other organs

Richard Lewis discusses the critical role pharmacists and health care providers play in ensuring safe administration of IVIG for patients with CIDP.

Thalassemia, a chronic blood condition that reduces hemoglobin levels, requires a hands-on approach from pharmacists in patient monitoring and diagnosis.

Circulating lactate is increased in patients with myelofibrosis and corresponds with the remodeling of lactate export channel monocarboxylate transporter 4, suggesting a link with fibrosis establishment.

BR55, an injection of perfluorobutane/nitrogen lipopeptide-coated microbubbles, could aid in the detection of angiogenesis and allow for earlier diagnosis in patients with Crohn disease.

Pharmacists should educate patients on abelacimab's potential therapeutic impact on atrial fibrillation patients at risk of stroke.

Proper nutrition and awareness of potential side effects from GLP-1 medications are essential for patients undergoing treatment.

Compared with other treatments for myasthenia gravis, nipocalimab demonstrated more significant improvements in disability scores, with tolerable safety.

The designation builds on previous regulatory action for ADI-001 and allows for expedited development of the treatment for systemic lupus erythematosus.

Donna Ryan discusses the landscape of GLP-1 medications and the lineup of indications that the drug class has received indications for in recent years.

More accurate prognostic risk assessment could aid pharmacists in the management and counseling of patients with primary myelofibrosis.

Often an indicator of fibrosis or damage to the heart, late gadolinium enhancement was found to be unaffected by the administration of intravenous immunoglobulin (IVIG) in pediatric patients with myocarditis.

Higher p16 expression in patients with sickle cell disease led to heightened risk of age-related complications.

Christian Ruff explains the strong efficacy associated with abelacimab compared with rivaroxaban in the AZALEA-TIMI 71 trial.

An inverse relationship was observed between genetic conditions associated with high levels of low-density lipoprotein cholesterol and the risk of developing T2D.

The approval marks the first subcutaneous infusion device approved for the treatment of Parkinson disease, providing a new option for patients with serious disease who are unresponsive to other therapies.

These results provide a pediatric lens into the potential risk of thromboembolic events in patients with dermatomyositis.

Myelofibrosis patients with CALR mutations have lower responses to symptoms and higher rates of anemia after 6 months of therapy with ruxolitinib.

Donna Ryan, MD discusses GLP-1 medications and new developments on the horizon, while providing some best practices for patients initiating treatment, including important nutritional and lifestyle changes,

Diagnosing anemia, a serious and common complication of patients with myelofibrosis, could be made easier with the use of red cell distribution width assessment.

Data indicates fedratinib’s role as an effective second-line treatment in patients with myelofibrosis who have been previously treated with a JAK inhibitor, such as ruxolitinib, though more research is necessary.