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 ...

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 ...

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 ...

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) ...

Signs and symptoms associated with ataxia and cognitive impairment, in particular, affected daily activities of the patients. Heterogeneity exists in the neurological manifestations exhibited by ...

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) ...

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 ...

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 ...

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 ...

Mycophenolate mofetil may have a role in alleviating liver injury secondary to drugs such as avacopan. An elderly female patient with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis ...

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