Progressive familial intrahepatic cholestasis (PFIC) is a rare group of inherited cholestatic liver disorders caused by defects in bile formation,...
Rare Disorders
Lymphatic malformations (LMs) are rare, congenital anomalies of the lymphatic system, which is part of the circulatory system and crucial...
Epidermolysis Bullosa (EB) is a group of rare genetic disorders characterized by fragile skin that blisters and tears from minor...
Duchenne Muscular Dystrophy (DMD) is the most common form of muscular dystrophy characterised by progressive skeletal muscle degeneration and weakness....
Spinal Muscular Atrophy (SMA) is a genetic neurodegenerative disease that leads to progressive muscle weakness and paralysis and if left...
Alagille syndrome (ALGS) is a rare genetic disorder that affects around one in every 30,000 live births. It results from...
Myasthenia gravis (MG) is a rare neuromuscular disorder that affects the transmission of signals between nerves and muscles. It is...
Pompe disease, also known as glycogen storage disease type II, is a rare genetic disorder that affects approximately 1 in...
Another gene therapy product has been added to the FDA-approved list: SKYSONA, a one-time gene therapy aimed to treat early,...
Today represents a key milestone for beta-thalassemia patients battling hereditary chronic disease. This comes after the approval that was granted...
