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.
compose your message
email sent successfully
Trusted Resources: News & Events
Latest announcements and gatherings
A Teenager’s Breakthrough Gene Therapy for Sickle Cell Disease
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.
Community CenterSuccessful Gene Therapy for Sickle Cell DiseasePrior to this report, the only curative ...
videos & visualsStepping Up: Calvanay’s advicehttps://www.youtube.com/watch?v=_x9Dhrim...
news & eventsBluebird Bio Presents Data From LentiGlobin Gene Therapy TrialBluebird Bio’s investigational gene th...
videos & visualsGene Therapy & Clinical Trials Webinarhttps://www.youtube.com/watch?v=yYexIUgH...
videos & visualsRoald Dahl’s Marvellous Children’s Charity “Everyone’s Business” campaignhttps://www.youtube.com/watch?v=VqdP3Sxa...
news & eventsVoxelotor can Promote Long-Term Benefits in Teens, Adults With SCD, Latest Clinical Data ShowGlobal Blood Therapeutics’ lead therap...
education & researchCollege Students Living with a Chronic ConditionThe term "chronic conditions" may apply ...
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.