DISCLAIMERThe information and materials accessed through or made available for use on any of our Sites, including, any information about diseases, conditions, treatments, or medicines, are for informational purposes only. The Content is not intended to be and is not a substitute for professional medical advice, diagnosis, or treatment, and your participation on our Sites does not create a healthcare professional-patient relationship. You should consult a doctor or other qualified health care professional regarding any questions you have about your health or before making any decisions related to your health or wellness. Call your doctor or 911 immediately if you think you may have a medical emergency.
Trusted Resources: News & Events
Latest announcements and gatherings
The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to IMR-687, Imara’s drug candidate for the treatment of sickle cell disease (SCD). The investigational therapy has also been granted orphan drug status by the FDA.
Rare pediatric disease designation is granted to drugs that show promise for rare and serious or life-threatening diseases in U.S. patients, primarily age 18 years or younger. The designation provides incentives to advance the development of rare disease drugs, including access to the FDA’s expedited review and approval programs.
“People living with sickle cell disease have limited treatment options for its devastating effects,” James McArthur, PhD, Imara’s president and CEO, said in a news release. “The FDA’s decision reflects its commitment to working with innovators and the patient community toward advancing safe and effective treatments for children suffering from the rare and damaging pediatric manifestations of the disease.”
news & eventsGeorgia Universities Join NIH-funded National Study of Bone Marrow Transplant for SCDThe Medical College of Georgia (MCG) and...
education & researchA Phase 3 Randomized Trial of Voxelotor in Sickle Cell DiseaseBackground: Deoxygenated sickle hemog...
news & eventsStem cell gene-editing method may be breakthrough for sickle cell researchResearchers have cured mice with a genet...
videos & visuals4 Transition Stages of Sickle Cell Disease and Tips for Each Stagehttps://www.onescdvoice.com/wp-content/u...
news & eventsApheresis Equipment Market is expected to reach USD 3.7 billion by 2024Highlights · The global apheresis eq...
education & researchShared Decision Making for Hydroxyurea Treatment Initiation in Children With Sickle Cell AnemiaClinical trials have demonstrated hydrox...
videos & visualsAdvances in the Treatment of Sickle Cell Disease: L-Glutamine, Crizanlizumab & Gene Therapyhttps://www.youtube.com/watch?v=wkm4axJ3...
send a message
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.
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.