The management of PI underwent a transformative shift in 1979 with the US Food and Drug Administration (FDA) approval of intravenous immunoglobulin (IVIg), which became the standard therapy for ...

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 term muscular dystrophy encompasses a group of genetic diseases characterized by progressive muscle weakness and wasting. There are different types of muscular dystrophy depending on genetic ...

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 ...

Overall, the life expectancy of patients with SCD is lower than that of persons without SCD. 1 However, life expectancy varies depending on the type of disease, treatments administered, and ...

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 ...

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 ...

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, life-threatening condition affecting hematopoietic stem cells. It is characterized by the premature destruction of erythrocytes, which ...

GISTs are fragile tumors with a tendency toward easy bleeding. Bleeding occurs due to pressure necrosis and perforation of the mucosal wall where the tumor develops, which causes a hemorrhaging of ...

Taylor Kane is executive director of Remember the Girls, a nonprofit group that advocates on behalf of females with X-linked disorders (Photo courtesy of Taylor Kane) New Jersey native Taylor Kane ...

Several factors influence the prognosis of gastrointestinal stromal tumors (GISTs), but the primary prognostic factors include the tumor staging and the tumor resectability. The staging for GISTs ...

Idiopathic pulmonary fibrosis (IPF) is an interstitial lung disease characterized by chronically progressing lung fibrosis. The most common symptoms of IPF include exertional dyspnea and chronic, ...