Duchenne Muscular Dystrophy (DMD) is the most common form of muscular dystrophy characterised by progressive skeletal muscle degeneration and weakness....
Rare Disorders
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...
Voxelotor (Oxbryta) is the first hemoglobin oxygen-affinity modulator. It has disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators...
What is Sarcoidosis? Sarcoidosis is a rare disease where inflamed cells clump together to make small lumps called granulomas which...
In this article, we will share with you the most important results of the American Hematology Society (ASH) oral poster...