Serum sickness is a rare but potentially severe complication of rituximab therapy. A patient with newly diagnosed progressive transformation of germinal centers (PTGC) associated with immunoglobulin ...

AI’s capacity to analyze and classify large volumes of data can be leveraged to enhance both diagnosis and management in Fabry disease. Artificial intelligence (AI)-based tools could improve the ...

A poor response to ITP therapy, characterized by corticosteroid resistance or dependence, was also found to be associated with chronicity. Factors predicting chronicity in immune thrombocytopenia (ITP ...

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 ...

In the case of Fabry disease, renal lysosomal inclusions were used as a surrogate biomarker that contributed to the approval of agalsidase beta. The role of biomarkers in diagnosis, severity ...

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 ...

ATTR-PN is an incurable disorder in which the achieving of meaningful improvement remains an important clinical goal. Hereditary transthyretin amyloidosis can result in significant morbidity; in ...

Clinicians changed their treatment plan in 19.4% of the paired pre- and post-MSDA time points, after reviewing the MSDA results. The multiple sclerosis (MS) disease activity (MSDA) blood test shows ...

Patients who were treated with inebilizumab had a greater reduction in the Myasthenia Gravis Activities of Daily Living scale and the Quantitative Myasthenia Gravis scale scores at week 26 compared to ...

Astragaloside II (AS-II) exerted its activity by suppressing β-catenin/inhibitors of DNA binding 2 (Id2)/myelin basic protein (MBP) signaling. Astragaloside II (AS-II) improves neurobehavioral ...

The tool addressed gaps in exposure to rare bleeding disorders often missing in traditional curricula. An online, case-based learning tool significantly enhanced trainee confidence and perceived ...

(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) ...