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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. Latest ULF News Check out the News Bulletin Section
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
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