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Meet Helen Obando, a shy 16-year-old who likes to dance when her body isn’t ravaged by the debilitating symptoms of sickle cell disease. The genetic blood disorder can cause strokes, organ damage and intense pain that can feel like glass running through her veins.
After a lifetime of pain and potential permanent damage to her body, Helen had the opportunity to receive a breakthrough experimental treatment at Boston Children’s Hospital that would make her the youngest person in the U.S. to have her DNA reset in an attempt to cure her sickle cell disease.
The outcome of her gene therapy could help determine how an estimated 100,000 people in the U.S. and millions more around the world are treated.
news & eventsFDA Clears Graphite Bio to Begin Trial for Gene Therapy in Sickle Cell DiseaseIn response to Graphite Bio’s investig...
news & eventsStatement on NHLBI Decision to Pause the Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Si...bluebird bio, Inc. suspended its clinica...
videos & visuals4 Transition Stages of Sickle Cell Disease and Tips for Each Stagehttps://www.onescdvoice.com/wp-content/u...
news & eventsExperimental Gene Therapy Reverses Sickle Cell Disease for YearsA study of an investigational gene thera...
news & eventsMARAC Advisory Statement: Gene Therapy & Bone Marrow TherapiesPlease note: A previous version of this ...
education & researchTransitioning your medical care: sickle cell diseaseSickle cell disease will be with you thr...
videos & visualsAdvances in the Treatment of Sickle Cell Disease: L-Glutamine, Crizanlizumab & Gene Therapyhttps://www.youtube.com/watch?v=wkm4axJ3...
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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.