Patients with MF and anemia had higher median annual all-cause healthcare resource utilization including inpatient admissions, outpatient visits, and emergency department visits compared to patients ...

In cases where it is not feasible to access brain biopsy for PCNSL diagnosis confirmation, a comprehensive examination of CSF becomes crucial. Cerebral spinal fluid (CSF) analysis with associated ...

Although generally safe, emicizumab should be used with caution, especially in patients with high cardiovascular risk. Emicizumab, a novel bispecific antibody mimicking factor VIII function, has shown ...

A barrier-free home environment and continuous caregiver support facilitated independence and improved quality of life. Achondroplasia, the most common skeletal dysplasia, and Chiari II malformation, ...

Growth velocity exceeded untreated reference values, and the 6-minute walk test showed functional gains in children treated with vosoritide. Children with achondroplasia experienced significant height ...

The targeting of factor XII activation is a key strategy in offering long-term prophylaxis to patients with hereditary angioedema. Garadacimab, a therapeutic targeting factor XII activation, has been ...

Plasma C4d and C4d/C4 ratio were significantly elevated in active AAV and decreased following induction therapy, suggesting their potential as biomarkers for disease activity. The classical/lectin ...

The improvement of local inflammation could be an effective clinical indicator of treatment success. Superficial swab cultures may not accurately identify the actual causative organism of systemic ...

Pretreating human astrocytes with interleukin (IL)-17A, IL-10, and IL-6 could help differentiate between serum obtained from anti-aquaporin 4 (AQP4) antibody-positive NMOSD and healthy controls.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews John Anderson, MD, chief research officer at AllerVie Clinical Research, on the complexities of diagnosing and treating ...

Those patients who had ever smoked had poorer lung function than patients who had never smoked and controls who had ever or never smoked. Alveolar volume analysis offers a simple and time-saving ...

In a clinical trial, vutrisiran was superior to placebo in reducing cardiovascular events and all-cause mortality in patients with ATTR-CM. The US Food and Drug Administration (FDA) has approved ...