Importance of Advocacy for Fabry Disease

Importance of Advocacy for Fabry Disease
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If you or a family member has a rare disorder such as Fabry disease, you may want to get involved with health advocacy efforts. In general, these efforts promote health and access to care at individual and community levels.

Here is more information about advocacy and ways in which it could help you.

Fabry disease

Fabry disease is a progressive rare genetic disease. It results from the abnormal buildup of a type of fat called globotriaosylceramide (Gb3 or GL-3) inside cells and tissues. Gb3 accumulation can cause a wide range of symptoms. These may include chronic pain, hearing problems, kidney damage, gastrointestinal and cardiac issues, and problems with eyesight.

Health advocacy

Health advocates support and promote patients’ healthcare rights. They also help build capacity to improve community health and enhance policy initiatives that are focused on available, safe, and quality healthcare.

Patient-centered care is a key goal of health advocacy. Other goals include safer medical systems and more patient involvement in healthcare delivery and design.

In terms of policy, advocates identify emerging public health issues that require action. They gather information on existing practices related to public health, monitor related legislation, and provide feedback on how certain issues affect communities. They also may help guide health policy reforms.

Advocates may act within organizations such as public health associations or patient organizations. Advocacy is typically carried out using multimedia platforms, political lobbying, and community mobilization.

Fabry disease advocacy groups

The National Fabry Disease Foundation supports the community through education, raising awareness about Fabry disease and diagnosis, providing assistance to patients, and advocating for disease issues.

There’s also the Fabry Support & Information Group. In addition to raising awareness about Fabry disease and its symptoms, the group advocates for community needs and supports efforts to find the best treatments and, ultimately, a cure for the disease.

The Canadian Fabry Association aims to increase awareness of Fabry disease and to improve the quality of life of patients and their families through research support, public education, and advocacy.

The Fabry International Network is a global network of patient organizations. It seeks to promote, through collaboration and communication, best practices to support those affected by the disorder.

Other organizations

The National Organization for Rare Disorders (NORD) is a nonprofit patient advocacy organization that works on behalf of people with rare diseases and the groups that serve them. In addition to organizing the U.S. portion of Rare Disease Day, NORD has advocated for healthcare services such as telehealth, or remote healthcare via telecommunications, to help people with rare disorders access healthcare.

Through an initiative that seeks to establish a Rare Disease Advisory Council in every U.S. state, NORD is also working to empower and equip members of the rare disease community to engage state leaders in matters important to patients and their families.

NORD’s “RareLaunch” training program hosted virtual workshops in 2020 aimed at empowering leaders — including caregivers, patients, and advocates — to start nonprofit organizations and research programs to help rare disease patients.

Eurordis is a similar advocacy group working for rare disease communities across Europe, and the organizer of Rare Disease Day.

Other efforts

Rare Disease Day takes place on the last day of February each year. Its main objective is to raise awareness among the general public and decision-makers about rare diseases like Fabry disease and their impact on patients’ lives. Advocacy takes center stage. Organizers may teach participants how to take action locally.

There’s also Rare Disease Week on Capitol Hill. This year, it will take place July 19–22, and bring together rare disease community members from across the country. The event is an opportunity to discuss federal legislative issues, meet other advocates, and share personal stories with legislators.

Elsewhere, discussions among scientists and patient advocacy groups about concerns of patients with Fabry disease and others regarding the current pandemic prompted a recent Rare Diseases Clinical Research Network survey online.

The federal government-run Rare Diseases Registry Program, called RaDaR, seeks to help patient advocacy groups with limited resources produce their own disease registries. Such registries are important in understanding disease development and characteristics. The Fabry Registry is the largest international patient registry database dedicated to the disease.

 

Last updated: Feb. 15, 2021

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Fabry Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”
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Özge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.
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Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”
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