leukodystrophyresourceresearch.org
Leukodystrophy Resource and Research Organisation Inc. | Useful Links
http://leukodystrophyresourceresearch.org/resources-news/useful-links
Leukodystrophy Resource and Research Organisation Inc. Australasian Leukodystrophy Foundation Incorporated No. IA41033. Donate and help us. How to fight Leukodystrophy. Resources & News. Grief & Wellness. Words for the New Year. ALDConnect Announcements 11.12.2015. ALDConnect Announcements – 10.22.2015. Gene Therapy — The time is now: Nick Leschly at TEDxBoston. Subscribe to our newsletter. Our newsletters are produced monthly. To join ALDConnect Patient Portal click here. Aidan has a Posse!
convergenceforum.com
About - Convergence Forum
http://convergenceforum.com/advisorseast.html
Life Science Leader Conference. News & Media. What opportunities get created as fields converge? What challenges surface as markets, policies, and investing dynamics shift? What new scientific discoveries are potential game-changers? Convergence exists to explore those questions (and many others), with a high-level audience interested in what’s happening in the rapidly-converging worlds of biotech, pharma, healthcare, medical devices, and the Internet. What Makes It Different. Our programs deliver a rapi...
starbeamstudy.com
Frequently Asked Questions | Starbeam Study
http://www.starbeamstudy.com/faq.php
Answers to Questions about CCALD, Gene Therapy. What is Childhood Cerebral Adrenoleukodystrophy. CCALD is a rare, fatal, genetic brain disorder. In its most severe form, it is characterized by a breakdown of the protective sheath around the nerves in the brain, called myelin, which surrounds and protects the nerve cells responsible for thinking and muscle control. As breakdown of the myelin occurs, the brain can no longer function properly. What is gene therapy. We have developed a Privacy Policy. To exp...
starbeamstudy.com
Childhood Cerebral Adrenoleukodystrophy | Starbeam Study
http://www.starbeamstudy.com/index.php
A clinical research study of an investigational gene therapy, has enrolled 18 boys, aged 17 and younger, who have been diagnosed with Childhood Cerebral Adrenoleukodystrophy. The goal of the Starbeam Study is to determine if the one-time investigational treatment can stop the progression of CCALD, and if it is safe and well tolerated. If your family member has CCALD, you may want to learn more about the Starbeam Study. About the Study Sponsor. In the Starbeam Study, gene therapy. Is being studied as a po...
starbeamstudy.com
Participation in Starbeam Study for ALD | Starbeam Study
http://www.starbeamstudy.com/study.php
The Starbeam Study, a clinical research study, has enrolled 18 boys, aged 17 and younger, who have been diagnosed with CCALD. In this investigational gene therapy. Study, a patient receives his own stem cells through autologous HSCT. GVHD and graft rejection do not occur with autologous HSCTs, and long-term immunosuppressive therapy is not needed. The goal of the Starbeam Study is to determine if the one-time investigational gene therapy. The sponsor of this study.
starbeamstudy.com
Starbeam Study » Site Map
http://www.starbeamstudy.com/sitemap.php
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.
starbeamstudy.com
About Adrenoleukodystrophy | Starbeam Study
http://www.starbeamstudy.com/ccald.php
About Childhood Cerebral Adrenoleukodystrophy. Symptoms of CCALD usually occur in a boy’s early childhood and may include hyperactivity, visual disturbances, auditory problems, impaired coordination, memory loss and seizures. Diagnosing CCALD can be difficult because initial symptoms are similar to other disorders, including attention deficit hyperactivity disorder (ADHD), autism, epilepsy and learning disabilities. In addition, many physicians are not aware of CCALD or may not look for it, because i...
starbeamstudy.com
Clinical Study Participation | Starbeam Study
http://www.starbeamstudy.com/participation.php
Learn About Clinical Research Studies. A clinical research study is a scientific evaluation to determine if and how a new medication or treatment may work. Through clinical research studies, doctors may identify new and improved ways to prevent, detect, diagnose, manage and treat different diseases. Clinical research studies may provide additional treatment options for people living with chronic conditions or other diseases. And The Center for Information and Study on Clinical Research Participation.
starbeamstudy.com
Clinical Study Resources | Starbeam Study
http://www.starbeamstudy.com/resources.php
Links to Helpful Websites for Caregivers. US Food and Drug Administration. Learn more about information and view resources on clinical research studies. National Institutes of Health. Read up on gene therapy in a comprehensive handbook on genetic research. The mission of ALD Connect is to improve health outcomes for individuals with X-linked adrenoleukodystrophy by raising awareness, empowering patients and accelerating the translation of scientific advances into better clinical care. National Organizati...
scdconference.com
About the Speakers
http://www.scdconference.com/about-the-speakers
Skip to Main Content. September 8, 2016 Four Seasons Hotel, New York, NY. This page contains links to third-party internet sites.*. Annual Sickle Cell Disease Therapeutics Conference was held on September 3, 2015. Please check back for 2016 conference information. Jonca Bull, M.D. - US Food and Drug Administration. Assistant Commissioner for Minority Health. US Food and Drug Administration. Sonja L. Banks - Sickle Cell Disease Association of America, Inc. Medical University of South Carolina. Robert Morl...
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