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 ...

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 ...

These estimates, despite certain limitations, offer a more accurate representation of the indolent SM burden and its comparison with other diseases. A recent study highlighted the significant burden ...

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 ...

Patients with CLL and a CSHA CFS score greater than 3 had significantly higher rates of treatment interruption due to adverse effects. The CSHA Clinical Frailty Scale (CFS) is a valuable tool for ...

(L-R) Rare Disease Advisor senior correspondent Larry Luxner, patient columnists Alithea Athans, Tara Keith and Tom Bartlett, and director of advocacy relations Vera Luxner (Photo by Riya Ajmera) ...

Bone marrow flow cytometry is one of the most efficient ways to diagnose or exclude PNH. A patient with a history of cortical vein thrombosis with new-onset pancytopenia was diagnosed with paroxysmal ...

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 ...

Researchers observed that sC5b-9 levels were proportional to the annual relapse rate after 1 year of follow up. Patients with neuromyelitis optica spectrum disorder (NMOSD) and high sC5b-9 levels ...

Researchers found that although primary intraosseous Rosai-Dorfman disease has histopathological features that overlap with IgG4-RD, it does not meet the criteria for IgG4-RD. Although primary ...

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, ...

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 ...