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
-
Development and Feasibility Testing of the Comfort Ability Program for Sickle Cell Pain: A Patient-Informed, Video-B...Objective: Even with optimized medical...
-
Orphan Drugs for Sickle Vaso-occlusion: Dawn of a New Era of Targeted TreatmentWhile an orphan disease in the USA, sick...
-
Gene therapies could transform the treatment of sickle cell diseaseManny Johnson keeps catching h...
-
FDA agrees accelerated approval pathway for GBT’s voxelotorGlobal Blood Therapeutics, Inc. ...
-
Bluebird bio acquires Durham, NC, manufacturing site for lentiviral vector productionbluebird bio said today it has acquired ...
-
GBT expands sickle cell disease pipeline with worldwide licensing agreement for inclacumab for the treatment of vaso...Global Blood Therapeutics, Inc...
-
Living With Sickle Cell Disease: One Person’s Story of Pain and Prejudice and Their Hopes for a Stem Cell TherapyMy name is Marissa Cors, I have sickle c...
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
SomeBodyToTalkTo connects people affected by rare diseases with trusted resources, supportive communities, and opportunities to share their experiences.