Feb 27 2014

Alexander Disease Research Update 2014 – LIVE WEBINAR

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Alexander Disease Research Update 2014 LIVE WEBINAR Please join Albee Messing, VMD, PhD, Professor of Neuropathology, Department of Comparative Biosciences, University of Wisconsin-Madison for his live webinar presentation on Saturday, March 8th, 2014 at 10:00 a.m. CST. Please click here to view the program information:  March 8 2014 Alexander Disease Update Webinar-140308-Combined Flyer

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Jan 10 2014

2013 Benefit Drawing

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The 2013 Annual Benefit Drawing Winners are: Mary Knapp from New Jersey won the one week stay at Orange Lake/Disney in Florida Donna Arnold from Michigan won the one week stay at Christmas Mountain in Wisconsin Dells, Wisconsin and Gloria LeDesma from Arizona won the handmade queen size quilt made by Anita Lewis.  

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Nov 01 2013

Starbeam Study Phase I Welcome Site

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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 A newer version of the Starbeam Study website will eventually be used to recruit study participants.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.

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Oct 30 2013

CADASIL Grant Instructions & Application

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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

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Oct 28 2013

bluebird bio Starbeam Study

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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

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Oct 28 2013

Augusto Odone Has Died

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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.

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Apr 13 2012

Benefit Raffle and Shire HGT MLD Clinical Program Update

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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

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Apr 13 2012

Social Security News

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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.

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Apr 13 2012

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Feb 09 2012

Dylan’s Dance

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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  

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