Learn About the
The Starbeam Study, a clinical research study of an investigational gene therapy, has enrolled 18 boys, aged 17 and younger, who have been diagnosed with Cerebral Adrenoleukodystrophy (CALD).
The goal of the Starbeam Study is to determine if the one-time investigational treatment can stop the progression of CALD, and if it is safe and well tolerated. If your family member has CALD, you may want to learn more about the Starbeam Study.
About the Study Sponsor
The sponsor of this study, bluebird bio, is a biotechnology company based in Cambridge, MA. bluebird bio is dedicated to developing next generation products based on the transformative potential of gene therapy to treat patients with severe genetic and orphan diseases. bluebird bio will be working with research centers of excellence to conduct the clinical study.
Exploring Gene Therapy for Treatment of Cerebral Adrenoleukodystrophy
Cerebral Adrenoleukodystrophy (CALD) is a fatal and rare genetic disorder that affects the nervous system of young boys. CALD involves a breakdown of the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control. Symptoms usually occur in early childhood and progress rapidly, if untreated, and can cause a vegetative state, ultimately leading to death.
In the Starbeam Study, gene therapy is being studied as a potential treatment for CALD. In the study, functioning genes will be used as medicine for the gene that is not working properly. Gene therapy has been studied as a potential treatment for genetic diseases since the 1980s. The goal of gene therapy is to help correct the genetic disease by providing a functioning copy of the gene to make up for the genetic defect.
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