Google
Trusted Resources: News & Events
Latest announcements and gatherings
In France, Boy Becomes First Sickle Cell Disease Patient to be Treated With Gene Therapy
A 13-year-old boy with sickle cell disease has become the world’s first patient to be successfully treated with gene therapy. The unidentified teenager remains free from sickle crises and other signs of the disease 15 months after his treatment, which was conducted as part of a Phase 1/2 clinical trial (NCT02151526).
The research team, led by Marina Cavazzana, MD, PhD, a hematology professor at Paris-Descartes University in France, inserted an antisickling β-globin gene, with the help of a viral carrier, into the boy’s blood-producing stem cells.
The report, “Gene Therapy in a Patient with Sickle Cell Disease,” appeared in the New England Journal of Medicine.
+myBinderRelated Content
-
Management of male fertility in sickle cell disease before and after hydroxycarbamideHydroxycarbamide (hydroxyurea or HU) is ...
-
FDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe SCD and Transfusion-Dep...BOSTON & ZUG, Switzerland--(BUSINESS...
-
Effort set to help sickle cell patients manage medsVanderbilt University Medical Center is ...
-
Updated Mechanisms Underlying Sickle Cell Disease-Associated PainSickle cell disease (SCD) is one of the ...
-
Novartis acquires US pharma research firm Selexys PharmaceuticalsSwiss-based pharmaceuticals giant Novart...
-
Sickle Cell Disease, Hydroxyurea: What You Should Knowhttps://www.onescdvoice.com/wp-content/u...
-
Sickle cell patients in UK survey, especially those 16 to 20, voice problems with care and pain relief givenIn a survey, patients across the...
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.