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Pharming Group is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. It is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines to serve the unserved rare disease patients. Its product includes Ruconest. The revenue is generated from the United States.
Stoke Therapeutics Inc is engaged in addressing the underlying causes of severe diseases by upregulating protein expression with RNA-based medicines. Using its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, it is developing antisense oligonucleotides (ASOs) to selectively restore protein levels. Its first compound, STK-001, is in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. The company primarily operates in the U.S.