Both patients werevanti-aquaporin 4 antibody-positive, based on which they were diagnosed with NMOSD. Researchers from Spain presented the cases of 2 patients with very late-onset neuromyelitis optica ...

(L-R) Christina Hartman, Kathryn Alexander, and Lesa Brackbill discuss newborn screening at the 2025 World Orphan Drug Congress USA in Boston, Massachusetts (Photo by Larry Luxner) Newborn screening ...

Scutellarein is a naturally occurring flavonoid compound derived from diverse sources. It shares antioxidant and anti-inflammatory properties with other well-known flavonoids. Scutellarein has been ...

Treatment-related adverse events were more frequent in the nipocalimab group than in the control group; however, they all were mild or moderate in severity. Nipocalimab, a monoclonal antibody with ...

VEXAS syndrome is known to be a mimicker of ANCA-associated vasculitis, making it a challenge to distinguish between the disorders. VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) ...

Last week, I had the incredible honor of speaking at the World Orphan Drug Conference (WODC), where I was invited to participate in a panel about the role of media as a lifeline in amplifying voices ...

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 ...

To improve outcomes, neonates with significant comorbidities such as sepsis and perinatal asphyxia on a background of FNAIT should be closely monitored. The prevalence of fetal and neonatal alloimmune ...

After 2 cycles of gemtuzumab ozogamicin, the patient achieved complete remission with clearance of leukemic and abnormal mast cells. A pediatric patient with refractory systemic mastocytosis (SM) ...

After initial treatment with a KIT inhibitor, hematopoietic stem cell transplantation was successful in resolving the symptoms. A new case has been reported in Pediatric Blood & Cancer of systemic ...

My hematologist has abandoned me. Now, I know that seems extreme, but it’s exactly how I felt when I heard the words, ‘I’ll be leaving in 2 months.” The dreaded words that no patient with a great ...

Which of the statements below about the symptoms of myelofibrosis (MF) are correct? Select one of the options for each question, then submit your answers to see how well you scored and how other test ...