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(Reuters) -The U.S. Food and Drug Administration has approved Ionis Pharmaceuticals' drug to treat a rare genetic disorder, making it the company's first wholly-owned drug, the health regulator's ...
Ionis Pharmaceuticals, Inc. is a biotechnology company based in Carlsbad, California, that specializes in discovering and developing RNA-targeted therapeutics.The company has three commercially approved medicines: Spinraza (), Tegsedi (), and Waylivra (Volanesorsen), and has four drugs in pivotal studies: tominersen for Huntington's disease (together with Roche), tofersen for SOD1-ALS, AKCEA ...
The U.S. Food and Drug Administration (FDA) on Thursday approved Ionis Pharmaceuticals and partner AstraZeneca's drug to treat nerve damage caused by a life-shortening rare disease. The drug ...
The results come as companies race to bring the first approved treatment for the disease and tap into a global market expected to surpass $16 billion by 2030, according to market research firm ...
The organization was founded by Stanley T. Crooke, former CEO of Ionis Pharmaceuticals and head of research at GlaxoSmithKline, in 2020. [3] The n-Lorem Foundation was established in January 2020 with the goal of taking advantage of the technology developed at Ionis to provide experimental treatments to patients suffering from rare genetic disorders.
IONIS-GCCR Rx, also known as ISIS-426115, is an antiglucocorticoid which is under development by Ionis Pharmaceuticals (formerly Isis Pharmaceuticals) for the treatment of diabetes mellitus type 2. [ 1 ] [ 2 ] It has also been under investigation for the treatment of Cushing's syndrome , but no development has been reported. [ 1 ]
The life-threatening genetic disease called hereditary angioedema causes unpredictable and frequent severe swelling of the skin, gastrointestinal tract, upper respiratory system, face and throat.
C. Frank Bennett is an American pharmacologist.Bennett is currently the Senior Vice President of Research and Neurology Franchise Leader at Ionis Pharmaceuticals. [1] He is a 2019 Breakthrough Prize winner in Life Sciences, which he shared with his collaborator Adrian R. Krainer for the development of an effective antisense oligonucleotide therapy for children with the neurodegenerative ...