“This is a great day for the Connecticut rare disease community! The Connecticut RDAC coalition would like to thank Governor Lamont, Lt. Governor Susan Bysiewicz, the Connecticut General Assembly Public Health Committee, especially Committee Chair Representative Jonathan Steinberg and Ranking Member William Petit for all their support and hard work on this legislation,” said Lesley Bennett, National Organization for Rare Disorders (NORD) Connecticut Rare Action Network Volunteer Ambassador. “This RDAC will give patients, families, caregivers, health care providers, advocates, researchers, and other stakeholders an opportunity to make formal recommendations to state agencies and our legislature on ways to develop public policy and health care legislation that will improve the lives of those impacted by a rare disease in Connecticut.”

NORD established Project RDAC in 2020 to increase the number of RDACs across the country and help optimize existing RDACs. Including Connecticut, a total of 23 states have signed legislation into law creating a Rare Disease Advisory Council, with RDAC legislation being signed into law in nine states since the start of 2021 alone.

“Sincerest appreciation to the coalition of lawmakers, patients, caregivers, and other members of the rare disease community who worked together to establish a permanent Rare Disease Advisory Council here in Connecticut,” said Peter Saltonstall, NORD President and CEO. “NORD has its roots in this state – through our founder, Abbey Meyers, and our founding nearly four decades ago. It’s incredibly empowering that Connecticut will have a permanent RDAC to help give its residents a stronger voice in their government and provide further partnership opportunities.”

Any condition that affects fewer than 200,000 Americans is considered rare. There are more than 7,000 known rare diseases, affecting 25-30 million Americans across a broad spectrum of medical conditions.  People living with rare diseases face many challenges, including delays in obtaining an accurate diagnosis, finding a health care provider with expertise in their condition, and a lack of affordable access to therapies and medications used to treat rare diseases.

For more information on NORD’s Project RDAC, visit rarediseases.org/projectrdac. To learn more about NORD’s policy work, visit: bit.ly/Policy-Issues.

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About the National Organization for Rare Disorders (NORD)

The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases in the United States. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. Since then, the organization has led the way in voicing the needs of the rare disease community, driving supportive policies, furthering education, advancing medical research, and providing patient and family services for those who need them most. Together with over 300 disease-specific member organizations, more than 17,000 Rare Action Network advocates across all 50 states, and national and global partners, NORD delivers on its mission to improve the lives of those impacted by rare diseases. Visit rarediseases.org.

The post Connecticut Establishes a Permanent Rare Disease Advisory Council appeared first on NORD (National Organization for Rare Disorders).

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Lanie advocates for those affected by… familial dysautonomia (FD)

Lanie is located in… New York, NY

How Lanie got here… I first heard of this rare disorder when a friend’s son was born with it 14 years ago. I was merely a donor to the Foundation until I was coincidentally recruited for the Executive Director position three years ago.

How Lanie’s previous work experience lends itself to her work in the rare disease community…  My entire career has been in fundraising, and I spent more than two decades as Director of Development for the hospice program in my community. While not a rare disease, during those years at hospice I learned a lot about working with families facing devastating health challenges and dealing with the loss of loved ones, as well as with building a sense of community around a common cause.

What the Familial Dysautonomia Foundation is hoping to accomplish this year… Like all nonprofits, when COVID-19 hit, all plans and projections for the year went out the window. Since March, we have faced canceled events and decreased donations. The key words during the pandemic seem to be “pivot” and “silver lining.” For example, we pivoted our annual conference from an in-person to a virtual event. The silver lining was that more people from around the country and around the world were able to join than ever before. We have also pivoted to find alternate sources of income to make up for losses as a result of the pandemic. Silver lining: a capacity-building seed grant from NORD.

How Lanie sees the Familial Dysautonomia Foundation changing in the next five years… The Foundation faces the challenge that its continued success portends its eventual demise. The Foundation funded research that, two decades ago, identified the gene that causes FD, leading to carrier testing and many fewer FD births. This means that with every loss, our community is shrinking. However, it’s important that we continue to ensure that communities at risk know about genetic testing. We also believe there may be many undiagnosed cases outside of the United States and Israel, where doctors have never seen or heard of familial dysautonomia. At the same time, there are a number of exciting new treatments in the research pipeline, and we are hopeful they will lead to substantially improved quality of life for people with FD in the very near future.

Lanie’s Dos and Don’ts for running an effective organization…  There are many constituencies involved in running an effective organization: patients, families, board members, physicians, researchers, staff, donors, etc. It is important for all voices to be at the table. Also, it is important to prioritize and stay focused on the big picture; otherwise, it’s too easy to get bogged down in the minutia.

Where Lanie developed her leadership skills… I think some leadership skills are part of one’s personality; other skills are learned and acquired, through a combination of training, education, mentorship and on-the-job experience.

How Lanie remains hopeful… I see the positive impact we are having on people with familial dysautonomia each and every day.

How Lanie is taking advantage of NORD member resources… I see NORD as a valuable resource and reach out to them often for information and guidance. NORD has provided me with scholarship access to its annual conference, the Rare Diseases and Orphan Products Breakthrough Summit. We recently were awarded a seed grant from NORD’s COVID-19 Rapid Response Leadership Series. I participate in many NORD webinars.

If Lanie found a cure for familial dysautonomia and had to find a new mission… There are so many other good causes out there, I would just find another one that needed my help!

The last book Lanie read is… The Vanishing Half by Brit Bennett

In her free time, Lanie enjoys… reading, exercising and cooking.

The post Head of the Herd: Lanie Etkind, Executive Director, Familial Dysautonomia Foundation appeared first on NORD (National Organization for Rare Disorders).

Interview with NORD Ambassador Maria Bellefeuille

The post Rare Action Network’s Maria Bellefeuille Interviewed by Patient Empowerment Network Leading up to Living Rare Forum appeared first on NORD (National Organization for Rare Disorders).