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 ...

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 ...

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 ...

Regarding the pediatric population, mold-active antifungal prophylaxis is recommended for pediatric patients at high risk of IFD, including a subgroup of children with ALL. Regarding the pediatric ...

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 ...

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 ...

Survival outcomes were similar with hypomethylating agents (HMAs) alone and an HMA plus venetoclax despite higher overall response rates. Investigators retrospectively evaluated 140 patients with TP53 ...

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 ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jennifer Miller, MD, a professor of pediatric endocrinology at the University of Florida in Gainesville who specializes in ...

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 ...

According to the physicians who responded to the survey, patients experienced a mean of 3.8 ± 9.8 months between the initial onset of EGPA signs/symptoms and their first consultation with a healthcare ...

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) ...