Ovarian cancer remains one of the leading causes of gynecologic cancer-related mortality worldwide. Although many patients initially respond to platinum-based...
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Progressive familial intrahepatic cholestasis (PFIC) is a rare group of inherited cholestatic liver disorders caused by defects in bile formation,...
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...
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...
The Food and Drug Administration (FDA) approved AstraZeneca's tremelimumab in combination with durvalumab and platinum-based chemotherapy for adult patients with...
The FDA approved the Investigational New Drug application for initiating a Phase 1/2 clinical study of YH001 in combination with...
The FDA has granted breakthrough therapy designation to the ROS1 inhibitor taletrectinib for the treatment of patients with advanced or...
The FDA has granted approval for pemigatinib (Pemazyre) to treat adult patients with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) and...
