Claseprubart incorporates YTE half-life extension technology, which is designed to enable infrequent subcutaneous self-administration. A phase 3 study is underway to evaluate the investigational ...

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 ...

IPF, the most common form of fibrosing interstitial lung disease (ILD), is characterized by abnormal accumulation of fibrotic tissue, causing thickening and stiffening of the lung parenchyma. 2 ...

As of May 2025, 62 active clinical trials are ongoing for Fabry disease, including 25 interventional and 37 observational studies, of which 8 are patient registries. Among the trials with known ...

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 ...

There were no significant differences in outcomes between low MFM-density states and fair or moderate MFM-density states, except for stillbirths, which were lower in moderate MFM-density states ...

Test Your Knowledge About the Genetics of Chronic Lymphocytic Leukemia Test Your Knowledge About the Prognosis of Chronic Lymphocytic Leukemia Test Your Knowledge About the Diagnosis of Chronic ...

In diagnosed cases of cutaneous mastocytosis, investigations to rule out systemic mastocytosis may be appropriate. A young child with a small chest lump and anemia was found to have cutaneous ...

Sickle cell disease is inherited in an autosomal recessive pattern, which means that a child is born with SCD only when they inherit 2 defective copies of the sickle cell gene (1 from each parent). If ...

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) ...

The CAPTIVATE trial met its target of at least 20 responders among the first 40 participants before all 40 completed part A. Dianthus Therapeutics announced an early “go” decision for claseprubart for ...

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 ...