For more information, please visit: www.Starbeamstudy.com This website is an interim site where parents and others can learn about the Starbeam Study and gene therapy. A newer version of the Starbeam Study website will eventually be used to recruit study participants. Therefore, it requires study site institutional review board approvals before it can be activated. The IRB-approved version of the website, which should be accessible before the end of the year, will then reflect that the study is open and enrolling participants.
Please click on the links below to view the instructions and print the grant application for submission, the application is due on or before February 1, 2014. Instructions: CADASIL GRANT APPLICATION INSTRUCTIONS Grant Application Form: CADASIL GRANT APPLICATION
Gene Therapy Study Seeking Participants with Childhood Cerebral Adrenoleukodystrophy An international clinical research study, called the Starbeam Study, is now enrolling boys, aged 17 and younger, who have been diagnosed with Childhood Cerebral Adrenoleukodystrophy (CCALD). CCALD symptoms usually occur in early childhood and progress rapidly, if untreated, ultimately leading to death. CCALD is a rare, genetic brain disorder that occurs when a broken gene, the ABCD-1 gene, does not work properly. Boys with CCALD do not produce any or enough of the adrenoleukodystrophy protein, or ALDP. Without enough ALDP, cells in the brain collect and store too many very long chain fatty acids. These fatty acids are harmful to brain cells and can trigger inflammation, which is responsible for the breakdown of myelin, a protective sheath that surrounds and protects the nerve cells. As the myelin breaks down, the brain can no longer function properly. When untreated, this
Augusto Odone born March 6, 1933 passed away on October 25, 2013 at the age of 80. A funeral service was held in Acqui Terma, Italy near Genoa on Saturday, October 26. Augusto, an Economist for the World Bank, was most known for his creation of Lorenzo’s Oil which was named after his son who died from Adrenoleukodystrophy at age 30. Lorenzo’s Oil lowers the very long chain fatty acids in the blood stream which are a major cause of the toxic effects of ALD.
The 2012 Benefit Raffle The 2012 Benefit Raffle winners are: -T. Henderson of Moss Point, Mississippi won a one week stay at Orange Lake Resorts in Kissimmee, Florida -M&D Massey of Jacksonville Beach, Florida won the king size quilt donated by Anita L. (donated in memory of her grandson Joseph Guzman) -E&M Monroe of Ashland, Wisconsin won a one week stay at Christmas Mountain Village in Wisconsin Dells. -J. Roth of Jefferson, Louisiana sold the most tickets! Thank you to everyone who helped make our benefit a great success!! Shire HGT MLD Clinical Program Update March 2012 Date: March 30, 2012 Dear MLD Patient Advocacy Organization: As you know, Shire Human Genetic Therapies, Inc. (Shire HGT) is sponsoring a clinical trial to evaluate the safety of an experimental intrathecal enzyme replacement therapy for patients with metachromatic leukodystrophy (MLD). The study (HGT-MLD-070) is designed for 40 weeks with
Social Security Announces New Conditions for Compassionate Allowances Program. There have been some Leukodystrophies listed. Michael J. Astrue, Commissioner of Social Security, today announced 52 new Compassionate Allowances conditions, Primarily involving neurological disorders, cancers and rare diseases. This Program fast tracks disability decisions to ensure that Americans with the most serious disabilities receive their benefit decisions within days instead of months or years. For more information on the Compassionate Allowances initiative, please visit www.socialsecurity.gov/compassionateallowances for the updated list and more information.
Featuring The Frank Palombi Orchestra and The Eddie Rodick Orchestra From Cleveland, Ohio Benefits “Dylans Quest For the Cure” Sunday, April 15th, 2012 Sygan S.N.P.J. Social Hall Doors open at 1:00 pm Music starts at 2:00 pm Admission is only $10.00 per person! Food and beverages available For more information, please call 412-889-6780 Dylan Changle, Born April 15th, 2009. Diagnosed with Leukodystrophy Krabbe Disease, September 1, 2009. Died: December 22, 2009
October 10, 2011 The University of Pittsburgh, Graduate School of Public Health, Department of Human Genetics, will be conducting a research stud to characterize the genetic mutations that cause Autosomal Dominant Leukodystrophy (ADLD). The results of this study will be important for the development of accurate genetic tests and thus will aid in the better diagnosis of ADLD patients. To learn more about this study and find out who can participate, view the attachment here.
February 14, 2011 If you or someone you know is a bone marrow, stem cell or cord blood transplant survivor, you hve vluable insights to share! Researchers at the Fred Hutchinson Cancer Research Center in Seattle, WA have received a grant from the National Caner Institute to see whether a website specifically designed for transplant survivors is a useful way to get current, helpful information to survivors across the country. The INSPIRE website is designed for survivors who are 2 or more years after transplant, healthy or not, because survivors have some special interests in maintaining their health. The website has specific tips and tools for: 1. Kepping you healthy nad monitoring for health issues (for you and your doctor) 2. Managing stress and worries, and making sure you enjoy life 3. Building energy Please consider taking part in this study which will help define better ways to deliver important information to