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Mission Statement: The United Leukodystrophy Foundation is dedicated to helping children and adults who have Leukodystrophy and assisting the family members, professionals and support services that serve them. The ULF is committed to the identification, treatment and cure of all leukodystrophies through programs of education, advocacy, research and service.

VISIT OUR:  LEUKODYSTROPHY SUPPORT COMMUNITY

The ULF has always encouraged a sense of family, support, belonging and communication. To further extend that philosophy, the ULF has partnered with the online community network Team Inspire to provide a mechanism for patients, caregivers and physicians to communicate in a common online forum for Leukodystrophy support. 

The Inspire community breaches a wide range of health related communities and felt that the ULF was a good fit for the community. The Inspire community provides several mechanisms for communicating with the members. There are blog pages where individuals can post their daily thoughts. There are discussion topics that members can start and get into a dialog with other members. There is also individual messaging capability to send messages to a single community member.  

We feel that the Inspire community is another positive step in that direction and we encourage everyone who can, to join the ULF Inspire community. Membership is completely free and it only takes a few moments to sign up and become a member. 

We hope that this will allow for a wider communication network and foster even more communication outside of ULF meetings and other conferences. Click the link above or on the left to get started now. 

2008 25th ULF Scientific Symposium and Family Conference

July 16th - 20th 2008

Senate Passes Historic Bill on Genetic Information Nondiscrimination

Americans Can Take Advantage of Health Advances without Fearing Discrimination

Washington DC– April 24, 2008 – With overwhelming support the Senate today passed by a vote of 95-0 the Genetic Information Nondiscrimination Act (S. 358).  With xx sponsors, the bill is a testament to a strong bipartisan effort. The Coalition for Genetic Fairness commends the members of the Senate for its commitment to affording comprehensive protections against genetic discrimination.
 
The Genetic Information Nondiscrimination Act (GINA) paves the way for the responsible use of genetic information while protecting against discrimination with respect to health insurance and employment.
 
“We are grateful for the bipartisan efforts of our sponsors in the Senate – Senators Edward Kennedy (D—MA) and Olympia Snowe (R—ME) as well as the tremendous support of Senator Michael Enzi (R—WY).  They are our champions and are making history today,” said Sharon Terry, President and CEO of Genetic Alliance, and President of the Coalition.  “Fears that genetic information could be misused hurts individuals, researchers, clinicians, and associated industries. Today, our fears have been addressed.”
 
Marla Gilson, Director of the Washington Action Office of Hadassah, said, “Just 10 years ago, only 100 genetic tests existed.  Today, that number has grown to over 1,000 and everyday these tests are helping diagnose thousands of health conditions.  Given the Jewish community's historical experiences with genetic issues, we worked hard to see that this bill was passed.”
 
The Coalition has worked for thirteen years toward the passage of legislation to eliminate the misuse of genetic information.  Discrimination on the basis of genetic information had led individuals to shy away from genetic testing that could help them manage their health proactively.  It also has caused many to opt out of clinical trials for fear that their genetic information would be used against them. This lack of participation has slowed the research and development of treatments and beneficial drugs.
 "We now have a huge task ahead of us.” said Kathy Hudson, director of the Genetics and Public Policy Center at Johns Hopkins University, “to make sure that doctors, researchers, and the public are aware of the new protections GINA provides."
Just as the House of Representatives did when it passed GINA in April 2007, with 224 cosponsors, S.358 protects Americans from discrimination by health insurers or employers based on genetic information by:
 
·     Prohibiting group health plans and issuers offering coverage on the group or individual market from basing eligibility determinations or adjusting premiums or contributions on the basis of genetic information.  They cannot request, require or purchase the results of genetic tests, or disclose genetic information.
 
·     Prohibiting issuers of Medigap policies from adjusting pricing or conditioning eligibility on the basis of genetic information.  They cannot request, require or purchase the results of genetic tests, or disclose genetic information.
 
·     Prohibiting employers from firing, refusing to hire, or otherwise discriminating with respect to compensation, terms, conditions or privileges of employment. Employers may not request, require or purchase genetic information, and may not disclose genetic information. Similar provisions apply to employment agencies and labor organizations.
 
The bill goes to the House, and is assured of passage there as early as next week.  The White House has signaled its willingness to sign GINA into law.
 

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The Coalition for Genetic Fairness is an alliance of advocacy organizations, health professionals, and industry leaders working to educate Congressional policymakers about the importance of legal protections for genetic information and ensure passage of meaningful genetic information nondiscrimination legislation.
 The Coalition for Genetic Fairness is led by: Genetic Alliance, Affymetrix, American Academy of Pediatrics, American Heart Association, American Society of Human Genetics, Brown University, Hadassah, National Society of Genetic Counselors, the National Workrights Institute and the PKD Foundation.

Coalition for Genetic Fairness • http://www.geneticfairness.org • 4301 Connecticut Ave. NW #404, Washington DC • 20008-2369 • Phone: 202.

Shire enhances its orphan drug pipeline with the acquisition of a new clinical candidate for Metachromatic Leukodystrophy

Basingstoke, UK and Cambridge, MA, US – 24 April, 2008 –

Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announces the acquisition of arylsulfatase –A (ASA) an Enzyme Replacement Therapy (ERT) in Phase 1-2 clinical trials for Metachromatic Leukodystrophy (MLD) from the Danish company Zymenex A/S (Zymenex).

MLD is a serious, life-limiting disease in which patients experience progressive irreversible neurological damage. MLD is caused by a deficiency in the enzyme ASA which causes an excess concentration of sulphatide in cells and an ensuing breakdown of myelin. There are approximately 2,000 MLD patients in developed world markets¹. The newly acquired ASA product, currently known as METAZYM™, has completed a Phase Ib clinical trial in 12 MLD patients in Europe and an extension to this study is ongoing. The product has received Food and Drug Administration (FDA) approval for its Investigational New Drug (IND) application to initiate a phase 2 clinical trial and has been granted Orphan Drug Designation in the United States and in the European Union. Sylvie Grégoire, President of Shire’s Human Genetic Therapies business, commented: “This product fits very well with Shire’s ERT portfolio of treatments for Lysosomal Storage Disorders (LSD). Shire HGT has been committed to MLD and by acquiring this mid-stage clinical program we hope to bring a MLD treatment to patients two years earlier than anticipated.” Shire is making a payment to Zymenex of US$135 million for the acquisition of global rights to the product upon completion of the transaction, which is conditional upon the receipt of customary consents. Zymenex is also providing certain transition services, including know how transfer, for up to six months after completion. The transaction includes no royalties or other downstream payment obligations. -ends-
¹ Scriver et al 1995

For further information please contact:
Investor Relations Cléa Rosenfeld (Rest of the World) +44 1256 894 160 Eric Rojas (North America) +1 484 595 8252
Media Jessica Mann (Rest of the World) +44 1256 894 280
Matthew Cabrey (Specialty Pharma - NA) +1 484 595 8248
Jessica Cotrone (Human Genetic Therapies - NA) +1 617 613 4640 Hampshire International Business Park
Chineham Basingstoke Hampshire RG24 8EP
United Kingdom
Tel +44 (0)1256 894000
Fax +44 (0)1256 894708
www.shire.com Press Release 2

Disease Background

Metachromatic Leukodystrophy (MLD) is in the family of lysosomal storage diseases (LSD’s). MLD is an autosomal recessive disease caused by a deficiency of the lysosomal enzyme arylsulfatase A (ASA) which results in an increased concentration of sulphatide in cells of the brains and in nonneural tissue, such as the kidneys and gallbladder. When these sulfated glycolipids accumulate in the brain, they cause a breakdown of myelin, a substance that protects the nerves in the brains and in the rest of the body. This breakdown is what makes MLD a progressive, neurodegenerative disease.

Symptoms and Patient Outlook

Sulfatide accumulation in the central and peripheral nervous system leads to destruction of the myelin sheath (demyelination). MLD has a spectrum of disease which can arise in infants and young children with a range of symptoms, though most are related to motor and cognitive decline. Most MLD patients are normal at birth but often die before age 20, with some patients within the first few years of life. During the final stages all patients reach a decerebrate, vegetative state. The majority of
cases are late infantile or juvenile patients. The number of adult patients exhibiting mild forms of MLD is unknown, as adult onset MLD can present with symptoms similar to psychosis.

SHIRE PLC

Shire’s strategic goal is to become the leading specialty biopharmaceutical company that focuses on meeting the needs of the specialist physician. Shire focuses its business on attention deficit and hyperactivity disorder (ADHD), human genetic therapies (HGT), gastrointestinal (GI) and renal diseases. The structure is sufficiently flexible to allow Shire to target new therapeutic areas to the extent opportunities arise through acquisitions. Shire’s in-licensing, merger and acquisition efforts
are focused on products in niche markets with strong intellectual property protection either in the US
or Europe. Shire believes that a carefully selected portfolio of products with strategically aligned and
relatively small-scale sales forces will deliver strong results.

For further information on Shire, please visit the Company’s website: www.shire.com.

"SAFE HARBOR" STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORM ACT OF 1995

Statements included herein that are not historical facts are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, Shire’s results could be materially affected. The risks and uncertainties include, but are not limited to, risks associated with: the inherent
uncertainty of pharmaceutical research, product development including, but not limited to the successful development of JUVISTA® (Human TGFβ3) and veleglucerase alfa (GA-GCB); manufacturing and commercialization including, but not limited to, the establishment in the market of VYVANSE™ (lisdexamfetamine dimesylate) (Attention Deficit and Hyperactivity Disorder (“ADHD”)); the impact of competitive products, including, but not limited to, the impact of those on Shire’s ADHD franchise; patents, including but not limited to, legal challenges relating to Shire’s ADHD franchise; government regulation and approval, including but not limited to the expected product approval date of INTUNIV™ (guanfacine extended release) (ADHD); Shire’s ability to secure new products for commercialization and/or development; and other risks and uncertainties detailed from time to time in Shire plc’s filings with the Securities and Exchange Commission, including Shire plc’s Annual Report on Form 10-K for the year ended December 31, 2007.