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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.

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Shire Human Genetic Therapies, Inc.

Progress Report on Enzyme Replacement Therapy Clinical Trial

August 20, 2009

Shire has an active development program in metachromatic leukodystrophy (MLD) and we are currently preparing to conduct a global phase II/III clinical trial with an experimental enzyme replacement therapy (ERT).  Shire is currently working very closely with the contract manufacturer, which is the only company to ever make this ERT, to produce this experimental enzyme for clinical trials.  Moreover, Shire is also evaluating all avenues to increase production capacity for this ERT, including the opportunity to bring manufacturing in-house. We plan to begin enrollment to the trial in the second half of 2009.

Shire is also working with regulatory agencies as well as individual medical institutions prior to initiating our phase II/III clinical trial.  A submission to regulatory agencies and approval by medical institutions of a trial protocol is standard industry practice prior to beginning any clinical trial.  This process is in place to ensure the protection of the safety and rights of the trial participants, and to help ensure that the trial design will address the questions that regulatory agencies might have regarding the experimental therapy.  Shire will post the details of the clinical trial, including entry criteria and locations, on www.clinicaltrials.gov, keyword MLD, after we have incorporated any changes and received the required approvals.

We understand that this trial creates great expectations for patients and their families and that our inability, at this time, to offer access to our experimental ERT to new patients outside of clinical trials may be disheartening and frustrating.  We at Shire are working diligently to bring a much needed therapy to MLD patients as soon as possible. 

In addition, Shire is committed to communicating in an open and transparent manner so that you can have appropriate expectations regarding access to this experimental ERT.

For any questions or clarification, please contact:

North America:

Lisa Marie Sturk, Ph.D.

Senior Medical Science Liaison

Tel:                   +1.612.259.7027

Toll-Free Fax:      +1.866.532.6770

Mobile:               +1.617.852.3696

Email:  lmsturk@shire.com

ROW:

Steve Moloney, MD

Senior European Medical Director

Tel:      + 44 (0) 1256 894508

Fax:     + 44 (0) 1256 894405

Mobile: + 44 (0) 7770 814107

Email: smoloney@shire.com

Global Medical Information:

Tiffany Crump, RPh

Medical Communications Manager

Tel:         +1.484.595.8850

Fax:        +1.484.595.8493

Email: TCrump@shire.com  

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. 

Genetic Alliance Issues Statement Applauding New NIH Therapeutics for Rare and Neglected Diseases Program
 
WASHINGTON – May 20, 2009 – Earlier today, the National Institutes of Health (NIH) announced the establishment of a new initiative called the Therapeutics for Rare and Neglected Diseases (TRND) Program, a federally mandated effort to encourage and speed the development of new drugs for rare and neglected diseases. Sharon Terry, president and chief executive officer of Genetic Alliance, a nonprofit health advocacy organization, issued the following comments in response to the establishment of TRND:
 
“Today marks a leap forward in transforming the rare and neglected disease drug development pipeline. NIH is a crucial entity working in this space. In the United States, a rare disease is defined as a condition affecting fewer than 200,000 people, and diseases lacking substantial therapeutic development activity are considered neglected diseases. Many genetic diseases are both rare and neglected, and collectively there are more than 6,000 rare and neglected diseases affecting over 25 million Americans. Fewer than 200 of these diseases have any therapy available, and progress in this area is limited within the current system. 
 
Clinical trial design is a unique challenge for rare and neglected diseases for reasons such as small patient populations and the need for global recruitment. Because of these challenges, it is critical to conduct effective trials from the onset, as subsequent opportunities may not be possible. Flexible, adaptive trial design and close interaction with the U.S. Food and Drug Administration will be especially important to consider in this context. In addition, engaging disease-specific groups in preparation for clinical trials to characterize the natural history of diseases and determine clinical trial endpoints is also essential and can begin right away through the application of existing tools, technologies and networks.
 
Genetic Alliance applauds the federal government for development of the TRND Program and views meaningful collaborations with external partners as a key element in the program. We have been collaborating with stakeholders in the health community including, disease-specific groups, umbrella organizations, and industry corporations while engaging in dialogue around regulatory and policy issues to bring about systems change in this area. This experience has shown us that collaborations through open and transparent processes are essential for change that will benefit health outcomes for all stakeholders.
 
We are excited for the potential the TRND Program holds and hope to collaborate with the program and its partners in system transformation.”

About Genetic Alliance – Genetic Alliance transforms health through genetics, promoting an environment of openness centered on the health of individuals, families, and communities. Genetic Alliance brings together diverse stakeholders that create novel partnerships in advocacy; integrates individual, family, and community perspectives to improve health systems; and revolutionizes access to information to enable translation of research into services and individualized decision making. For more information about Genetic Alliance, visit http://www.geneticalliance.org.