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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 ...
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 ...
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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 ...
Ultragenyx Pharmaceutical Inc. is an American biopharmaceutical company involved in the research and development of novel products for treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and high unmet medical need. The company works with multiple drug modalities including biologics, small ...
Nusinersen, [7] marketed as Spinraza, [4] is a medication used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder. [8] [4] In December 2016, it became the first approved drug used in treating this disorder.
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 FDA grants orphan drug and rare pediatric disease designations to Ionis' (IONS) ION582 for treating Angelman syndrome. These designations will help incentivize development of the candidate.