Progressive familial intrahepatic cholestasis (PFIC) is a rare group of inherited cholestatic liver disorders caused by defects in bile formation,...
Rare
Duchenne Muscular Dystrophy (DMD) is the most common form of muscular dystrophy characterised by progressive skeletal muscle degeneration and weakness....
Myasthenia gravis (MG) is a rare neuromuscular disorder that affects the transmission of signals between nerves and muscles. It is...
A new hope is looming for Myelofibrosis patients who experienced a suboptimal response to ruxolitinib and those who were JAK...
Emirates Oncology Society (EOS) and Ipsen joined forces to raise awareness of Neuroendocrine Tumors (NET), a rare type of cancer....
Several indications and molecules were approved in July by FDA including the following: Asparaginase erwinia chrysanthemi (recombinant)-rywn) as a component...
What is Wilson's disease? Wilson's disease is a rare genetic autosomal disease. It occurs in approximately one in 30,000 to 40,000...
Calciphylaxis is an uncommon, rare, serious, and fatal disorder that is characterized by: Calcification of the small blood vessels...
Char syndrome is a rare, genetic, multiple congenital anomalies/dysmorphic syndrome characterized by: Triad of patent ductus arteriosus (PDA) Typical facial...
Anaplastic large-cell lymphoma (ALCL) is a rare type of non-Hodgkin lymphoma that T-cells. Pathologically, T-cells appear abnormal, large, and undeveloped. Most patients are...
