Promising results from a preclinical experiment have the potential to modify how myelofibrosis is treated in the future.
Studies demonstrate that alpha-1 antitrypsin deficiency does not predispose a never-smoker to lung cancer.
The PIGA gene mutation alone does not explain the pathophysiology of PNH. The clonal expansion of PNH cells remains a mystery.
The nation’s first CureDuchenne Clinic has opened in a suburb of Dallas, Texas, to treat Hispanic patients with rare neuromuscular diseases.
The American Society of Hematology (ASH) is establishing a nationwide learning community to improve the care and quality of life of people with SCD.
Untreated Wilson disease can eventually result in death, but patients who receive adequate treatment can have a normal lifespan
Prominent pulmonologist Paul W. Noble, MD, says zinc may be the key to treating and even reversing idiopathic pulmonary fibrosis.
Researchers described the case study of a patient with vaccine-induced prothrombotic immune thrombocytopenia.
The incidence of many rare diseases is rising rapidly, especially in the Western world, and advancements in therapeutics have not quite caught up. In the United States, for example, billions of dollars are being spent annually for treating autoimmune disorders and conducting trials to find future solutions. Immune thrombocytopenia (ITP) is one such disease; it…
Studies indicate that patients with ANCA-associated vasculitis have a heightened risk of developing cardiovascular disease.