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 ...

The global prevalence of PNH is as high as 15.9 individuals per 1 million people. The annual global incidence of PNH is around 5 to 6 individuals per 1 million people. 5,6 In the United States, ...

The use of semaglutide has been associated with improvement of treatment-resistant mast cell activation symptoms in an overweight patient with SM. Glucagon-like peptide-1 receptor agonists (GLP-1RAs) ...

In a phase 3 trial, participants taking venglustat demonstrated statistically significant improvement in neurological symptoms compared with those receiving ERT. Sanofi announced that the US Food and ...

Gastrointestinal involvement in systemic mastocytosis (SM) is a common complication and often leads to endoscopic findings. Gastrointestinal (GI) symptoms are the second most frequent clinical ...

In the ADAPT SERON trial, treatment with efgartigimod led to rapid, significant, and sustained improvements in symptoms such as speech, vision, swallowing, and physical function. The US Food and Drug ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Albert Faro, MD, vice president for clinical affairs at the Cystic Fibrosis Foundation (CFF). Dr. Faro speaks about COVID-19’s ...

Consolidation or maintenance therapy significantly improved progression-free survival and overall survival. Zanubrutinib, lenalidomide, temozolomide, and rituximab (RLZT) combined with or without high ...

Treatment with obexelimab compared with placebo was associated with a significantly longer time to the first IgG4-RD flare that required the use of rescue therapy (P .001). One hundred ninety-four ...

Emerging therapies such as bezuclastinib and elenestinib show promise in improving disease control with potentially fewer toxicities. Other novel strategies, such as immune-based therapies, are under ...

Relative survival increased from 64% to 66%, overall survival from 54% to 55%, and lymphoma-specific survival from 64% to 66%. The advent of chimeric antigen receptor T-cell (CAR-T) therapy for ...

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...