The global Alagille Syndrome Treatment Market focuses on therapies aimed at managing the symptoms and complications of Alagille Syndrome (ALGS), a rare, multi-system genetic disorder caused primarily by mutations in the JAG1 gene. ALGS is characterized by abnormalities in the liver, heart, eyes, skeleton, and kidneys, with the most common and severe manifestation being chronic cholestasis...

The global Charcot-Marie-Tooth Disease Market focuses on the management and development of therapies for CMT, which represents a group of inherited neurological disorders that damage the peripheral nerves, leading to muscle weakness, loss of sensation, and physical impairment, predominantly in the feet, legs, hands, and arms. Currently, the market is characterized primarily by supportive and...