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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 ...
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 life-threatening genetic disease called hereditary angioedema causes unpredictable and frequent severe swelling of the skin, gastrointestinal tract, upper respiratory system, face and throat.
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 ...
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.
Tofersen was developed by Ionis Pharmaceuticals and was licensed to, and co-developed by, Biogen. [7] [8]The effectiveness of tofersen was evaluated in a 28-week, randomized, double-blind, placebo-controlled clinical study in 147 participants with weakness attributable to amyotrophic lateral sclerosis and a superoxide dismutase 1 (SOD-1) mutation confirmed by a central laboratory. [2]
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.