As the new President of the Dysautonomia Foundation, I'd like to introduce myself and describe the current state of the Foundation's activities and our plans for the future.

For the last fifteen years I have been involved with the Dysautonomia Foundation: first (and always) as a parent of a child with FD, then as a member of the Board of Directors, and for the last several years, as a Vice President of the Foundation. Now, it is my great privilege and honor to serve the Foundation as its newest President.

The work that lies ahead is challenging, but I start my tenure with the knowledge that the Foundation's two most recent past presidents, Jennifer Sonenshein and Jeffrey Goldberger, have made great strides in pursuing the Foundation's mission to serve the FD population by providing outstanding medical care, research, public education and support services for the benefit of all FD patients and their families.

Certainly, there remains much work to do, and the Foundation is poised to not only continue its good work, but to expand the range and depth of its mission.

However, before laying out our plans for the future, it is useful to look back on our history as well as some of the major accomplishments that have shaped our "FD world," so that we can set a course for the future that is most beneficial to our FD community.

In terms of FD history, I'd like to point out three of the most important accomplishments over the last several decades:

1. The Foundation was created over fifty years ago by concerned parents who had nowhere else to turn. This "strength in numbers" led to the single most important factor in the care of FD patients: the establishment of dedicated treatment facilities (in the US and Israel) that serve as centers of excellence for medical care specifically for FD patients. These centers remain the only such facilities in the world, and have been used by more than 90% of all FD patients, worldwide.

2. The establishment of a permanently endowed professorship, at NYU, in the study of FD so that research and clinical care would have a worldwide focal point. This single accomplishment has led to the centralized coordination of care and research for FD, and has facilitated the work of the world's foremost authority on FD, Dr. Felicia Axelrod.

3. The funding of independent research initiatives that led to the understanding of how FD functions and, even more importantly, the funding of a decade of work to lay the groundwork for, and ultimately to facilitate the discovery of the FD gene. All recent discoveries and therapies for FD are based on this work that has been funded by the Foundation.

As a result of the decades of work that produced these accomplishments, our "FD world" is now shaped by three exciting factors, each of which reflects our progress and points the way to new challenges:

1. FD patients live longer. This decrease in mortality and increase in life expectancy is the direct result of better treatment and research focused on FD. While we should all be grateful that FD is no longer considered a 100% fatal, childhood disease, we now have new challenges in addressing the needs of adult FD patients and further reduction in the mortality and morbidity of the disease for all FD patients.

2. Discovery of the gene has led to new research initiatives focused specifically on genetic treatment and cure. Researchers can now focus on addressing the root causes of the disorder, finally opening the door to the pursuit of definitive treatments that will reduce symptoms, stop the degenerative effects of the disease and, hopefully, lead to a cure.

3. Genetic screening has the potential to reduce the number of new cases of FD. The discovery of the gene has led to a definitive carrier test. We can now screen couples to determine if the risk of having a child affected by FD is present, and by doing so, we have the opportunity to decrease the frequency of new FD cases. Of course, these goals can only be achieved through a massive outreach to the Ashkenazi population to "get tested."

It is these three factors that influence the Foundation's current mission. Each one of the factors shapes our goals:

1. Factor: Patients live longer. Over the last twenty years, FD patients, under the care of the treatment centers, and through the use of gatrostomy, fundoplication, medication and other therapies, live significantly longer and are much less likely to die from aspiration pneumonia. As a result, the percentage of FD patients who are adults has increased dramatically. But they continue to require significant medical care as well as social services, and they are still subject to other causes of mortality. If we have succeeded in helping FD patients to live longer, we must now focus on helping them to live better lives. 
 
Goals:

• Provide enhanced adult patient medical services in addition to existing pediatric services. 

• Provide directly, or in conjunction with other organizations, social and psychological services for all FD patients. 

• Fund new research into the causes and prevention of FD-related deaths. (In the past we focused on reduction of mortality, and this led to treatment that reduced deaths from aspiration pneumonia. We now need to focus research on other causes of death to further reduce FD mortality.)

2. Factor: Discovery of the gene has led to the opportunity to study the root causes and mechanisms of FD. Such studies could lead to significant advances in treatment and, ultimately, could lead to a cure for FD. 

Goals: 

• Continue and expand funding into research specifically focused on gene therapy and other methods that directly affect the expression and progression of FD symptoms. 

•  Continue to encourage other organizations (such as the NIH) to pursue their own initiatives into the study and funding of FD-related research.

3. Factor: Genetic screening throughout the Ashkenazi population could significantly reduce the frequency of new FD cases. Yet we must not lose sight of the fact that the current patient population will require medical treatment and other services on an on-going basis. 

Goals:

• Large-scale public awareness initiatives must be undertaken to get our message out to both the medical community and the Jewish community.

• Our message is a dual message: We must raise public awareness that a) the treatment centers are available to anyone with FD, and b) new cases of FD can be prevented through public education of genetic screening.

To sum up - the Foundation has been the leading organization in the pursuit of services, treatment and research for the FD community for the last fifty years. Going forward we will try to uphold and build upon that tradition by supporting the following initiatives:

• Medical Treatment for existing and new patients; 

• Adult patient services, including social and psychological services; 

• Research into the causes and prevention of FD patient mortality; 

• Research into genetic and other medical therapies leading to the eradication of FD symptoms and a cure for FD; 

• Public awareness campaigns directed at the medical community and the public at large, geared toward preventing the occurrence of new cases of FD; 

• Public awareness campaigns geared toward informing the public of the resources available for treating FD patients; 

• Advocacy for legislation that provides benefits and services to FD patients and their families through new and existing government programs.