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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 ...
(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 CEO Brett Monia said the company estimates that only 20% of the patients suffering from ATTR-PN are on treatment. "This is a market where more than one player can have great success," he added.
The company maintains its top-and bottom-line guidance for 2021. Ionis (IONS) incurs wider-than-expected Q3 loss. The company maintains its top-and bottom-line guidance for 2021
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]
Silence Therapeutics (Nasdaq: SLN [1]) is a London-based pharmaceutical company formed in 1994. The company has pioneered the development of short-interfering ribonucleic acid (siRNA) therapeutics for the treatment of rare diseases.
Ionis (IONS) incurs a narrower-than-expected loss in Q3. Its sales also beat estimates. Skip to main content. Sign in. Mail. 24/7 Help. For premium support please call: 800-290 ...
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.