The expression of AQP4 In the respiratory epithelium suggests that lung involvement might be an unrecognized feature of NMOSD. Patients with neuromyelitis optica spectrum disorder (NMOSD) should be ...

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 ...

Elafibranor is currently under regulatory review by the European Medicines Agency and the UK Medicines and Healthcare products Regulatory Agency. The US Food and Drug Administration (FDA) has granted ...

Results showed a significantly higher prevalence of liver disease among patients with chronic pruritus compared to healthy controls. Patients with liver diseases of different etiologies, including ...

Over 80% achieved a clinically meaningful improvement in at least one of the scales assessed. Treatment with risdiplam over 30 months led to overall clinically meaningful improvement in most adult ...

The prevalence of AATD in the offspring of parents with AATD in our study was 37%. A risk of pregnancy complications and adverse birth outcomes has been observed in mothers with alpha-1 antitrypsin ...

In their study, the investigators found that 8 of the 61 participants had 1 or more of the following conditions: AAV, non-AAV, autoimmunity, immunodeficiency, and malignancy. Follicular bronchiolitis ...

The severity of clinical manifestations in CAD can vary depending on the specific type of cold-sensitive antibodies involved. While cold agglutinin and Donath-Landsteiner antibodies recognize antigens ...

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 ...

Castleman disease and IgG4-RD have overlapping disease manifestations that can complicate the diagnostic process. A male patient initially suspected of having immunoglobulin G4-related disease ...

The novel anti-IL-36 receptor monoclonal antibody recibokibart demonstrated favorable safety and efficacy in treating patients with acute generalized pustular psoriasis (GPP) in a phase 1b study ...

The Collaborative Trajectory Analysis Project seeks to broaden patient eligibility for clinical trials in Duchenne muscular dystrophy. Larry Luxner, senior correspondent for Rare Disease Advisor, ...