Shop Online While Giving to ULF:

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

NIH Undiagnosed Study

 The National Human Genome Research Institute at the National Institutes of Health (NIH) is sponsoring a study that seeks to learn more about how parents of children with an undiagnosed medical condition think and feel about their child's condition. We hope that this knowledge will improve the health care and counseling for these parents. Men and women who are 18 years or older and have at least one child with a medical condition that has remained undiagnosed for more than 2 years are needed to take part in this study. Participation involves one survey that takes about 45 minutes to finish. The survey can be taken online or a paper copy can be mailed to you. For additional information about this study, you may review the Notice to Participants disclosure.

Research Study: You are invited to participate in a research study sponsored by the National Human Genome Research Institute, one of the National Institutes of Health.

Why is this study being done: To lean more about how parents of children with an undiagnosed medical condition think and feel about their child's condition.

Who can be in this study? One survey that takes about 45 minutes to finish. The survey asks for your thoughts and feelings about having a child or children with an undiagnosed medical condition. The survey als asks questions about your everyday life.

What are the risks of the study? There are no physical risks of taking part in this study. Some of the topics covered in the survey are sensitive or personal. There is a risk that taking the survey may cause you to become concerned or upset. If this happens, please contact Anne Madeo (her contact information is listed below) so that she may help you. If you are worried about the chance that you may become upset, you do not have to take this survey.

Are there benefits to taking part in the study? No, there are no personal benefits to you from taking part in this study. However, your answers would tell us more about the experiences of parents who have a child or children with an undiagnosed medical condition. We hope that this knowledge will improve health care and counseling for these parents.

Will I be paid? No, you will not be paid for participating in this study.

Do I have to take part? No, you do not have to be part of this study if you do not want to. You can stop taking the survey at any time, or skip any questions(s), without penalty.

Who else will know that I am in the study? No one else will know that you are in the study. The study is completely anonymous. We do not ask for your name on thsi survey. No researcher will ever have access to your name. This study will not be part of any medical record or medical visit. When we report our research results, it will be done anonymously so that you will not be identified.

How do I take part? The survey can be done online or though the mail. If you would like to fill out the survey online, please go to:

www.surveymonkey.com/SurveyExperienceUncertainty

The password to enter the survey is:

Uncertainty

If you would like to fill out the survey on paper rather than online, contact Anne Madeo at the contact information listed below. She will mail you a copy of the suvey along with a stamped return envelope. Any contact information you give the researchers so they can send you a survey will be destroyed after it is mailed to you and will not be linked to your responses.

How can I learn about the results of this study? After the study is completed, we plan to post a short summary of our results on the support network websites or listervs where you learned of this study. Please check the website in the future for this information.

Problems or Questions? If you have any problems or questions about this study or about your rights as a participant, please contact Anne Madeo. If you have any concerns about your child's condition or about issues brought up by this survey, please contact Anne Madeo.

If you have questions, please contact:

Anne C. Madeo, MS, Prinicipal Investigator

Genetic Counselor

National Human Research Institute

National Institutes of Health

Bldg. 31, room B1B36

31 Center Drive, MSC 2073

Bethesda, MD 20892-2073

Phone: 301-443-2635

Email: anne.madeo@nih.gov

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.