Google
Trusted Resources: News & Events
Latest announcements and gatherings
Potential treatment targeting nitric oxide levels, olinciguat, named orphan drug by FDA
The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to olinciguat (IW-1701) as a potential treatment of sickle cell disease (SCD), Ironwood Pharmaceuticals announced.
Evidence suggests that the symptoms of sickle cell disease may be associated with nitric oxide deficiency in the blood. Nitric oxide is a free radical produced by several cells and used as a signaling molecule that works to induce cellular events that promote blood flow. Patients with sickle cell anemia have a reduced bioavailability of nitric oxide.
Olinciguat is an oral investigational therapy that stimulates an enzyme called soluble guanylate cyclase (sGC), known to play a key role in the production of nitric oxide.
The stimulation of sGC can restore the bioavailability of nitric oxide in the blood to increase blood flow, which may stop the destruction of blood cells and help address disease symptoms.
+myBinderRelated Content
-
Orphan Drugs for Sickle Vaso-occlusion: Dawn of a New Era of Targeted TreatmentWhile an orphan disease in the USA, sick...
-
Scientists edit genome to cure sickle cell anemiahttps://www.youtube.com/watch?v=4LKzM3G6...
-
Transcutaneous electrical nerve stimulation (TENS) for pain management in sickle cell diseaseAs defined by the American Physical Ther...
-
Stuart H. Orkin, MDDr. Orkin is David G. Nathan Distinguish...
-
Sickle cell patient with severe anemia rapidly improves with voxelotor, case study showsGlobal Blood Therapeutics (GBT) reported...
-
Ironwood Pharmaceuticals Announces FDA Orphan Drug Designation for Olinciguat for the Treatment of Sickle Cell Disea...Ironwood Pharmaceuticals, Inc., a commer...
-
ASH launches sickle cell disease clinical trials network to accelerate therapy developmentASH launched a clinical trials network d...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Powered by
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.