Ivabradine treatment slowed resting heart rate and reduced the levels of N-terminal prohormone of brain natriuretic peptide (NT-proBNP) in some patients with ATTR. MINNEAPOLIS—The sodium channel ...

In the pivotal LUNA 3 trial, durable platelet response at week 25 was achieved by 23% of patients with rilzabrutinib compared with 0% with placebo. The US Food and Drug Administration (FDA) has ...

The potential of vitamin D as an add-on therapy lies in its immunomodulatory effect on T cell-mediated and humoral immune responses, complement activation, cytokines, and inflammasomes. Vitamin D ...

Which of the statements below about therapies for idiopathic pulmonary fibrosis (IPF) are correct? Select one of the options for each question, then submit your answers to see how well you scored and ...

Several guidelines have been developed for the diagnosis and management of Fabry disease. The focus of treatment is preserving organ function, maximizing quality of life, and normalizing life ...

As aspirational goals for the care of patients with PBC move toward liver test normalization, adjunctive therapy appears promising. The triple therapy combination of ursodeoxycholic acid (UDCA), a ...

Recurrent cutaneous swelling, which was the most commonly reported symptom, occurred in all 22 of the study participants who underwent evaluation. Our global understanding of hereditary angioedema ...

Analysis of HLA, HPA, and CD36 antibodies, which are found in FNAIT and immune-mediated platelet transfusion refractoriness, is critical for minimizing an individual’s risk for alloimmunization. The ...

Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...

Circulating biomarkers suggest the presence of a pubertal growth spurt in affected adolescents. In children with achondroplasia, circulating CXM levels were significantly associated with annualized ...

A tailored anesthesia management plan is necessary for pregnant women with SM undergoing elective cesarean delivery to help guide clinicians in optimal patient care. A theoretical plan for anesthetic ...

Dr. Hirano has seen about 50 patients with TK2d and is currently treating 26 of them. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews neurologist Michio Hirano, MD, of Columbia ...