Continuity of Rare

July 9, 2020

“Somebody else probably needs it more than me.”

When Todd Talarico asked his community the question, “How can we help?” most families were slow to accept financial support. However, as testimonies of crippling financial and emotional burdens placed on families due to COVID-19 are being shared across the nation, it has become clear that the needs of rare disease foundations and their communities have never been greater.

In an effort to empower advocates and rare disease thought leaders, Global Genes has distributed nearly $200,000 to date to 15 incredible RARE Foundation Alliance members for the Continuity of Care RARE Patient Impact Grant. These organizations, while diverse in the communities they serve, have all faced similar challenges due to COVID-19. They are passionate and unrelenting in their crusade for improving the lives of their communities, and truly mobilize where there is need.

“Having a rare disease is already isolating, but COVID-19 has amplified that. For some people, their entire social life was basically gone overnight.” -Jennifer Knapp, Adrenal Insufficiency United

Empowering Connectedness

One of the motivations for creating the Continuity of Care RARE Patient Impact Grant is to help with the emotional and psychosocial burden placed on the rare community during COVID-19. Cher Bork of the Alagille Syndrome Alliance (ALGSA) says continued, if not increased connection among their community members has been crucial over the past few months.

“We are missing out on social interactions. Some parents aren’t even able to interact with their own families. We have one mom who is a nurse and is forced to sleep in the garage to protect her child. Others are experiencing job loss and having to step in as an at-home teacher and therapist with little support.” -Cher Bork, Alagille Syndrome Alliance

Other organizations have also increased their virtual communication in an effort to remain connected. “We try to make ourselves and our community available to provide emotional support… And we try to communicate things other than sounding the alarm of COVID, which we’re seeing everywhere else right now,” explains Todd Talarico of the APS Type 1 Foundation.

Both APS Type 1 and Alagille Syndrome Alliance have begun organizing regular group zoom calls with their communities, allowing patients to talk about the challenges they’re facing, as well as connect with industry moderators who provide updates on research progress and disease-specific support as it relates to COVID-19.

Other organizations like Fighting for Kaiden and GRIN2B Foundation are staying connected  through online Facebook groups, and have even begun to revamp their websites in order to reach more patients internationally. Without the ability to meet face-to-face, providing tools for connectedness to members can be extremely impactful.

Beyond the Emotional Toll

Additionally, Jennifer Knapp of Adrenal Insufficiency United (AI United) says, “Because people who are adrenal insufficient suffer from a lack of cortisol, some who experience emotional trauma, anxiety, and depression from the stress of COVID-19 have more day-to-day challenges as they manage their medication.” For those who are adrenal insufficient, the emotional toll of distancing can also have a physical effect on their health.

Jennifer says that support for Telehealth services, including occupational therapy sessions and counseling, is where AI United is seeing the most need in their community. They plan on providing families with resources to purchase laptops and iPads, and are working to provide support for families who are also tasked with homeschooling and basic living expenses.

“I didn’t realize how little certain school districts were able to do to help these families. Our kids need a lot of support, they really need consistency with their therapies, their teachers and their routine to thrive.” – Liz Marfia-Ash, GRIN2B Foundation

Liz Marfia-Ash of GRIN2B Foundation and her team are focusing on coming up with creative ways to support therapies no longer offered in-person to their patients with the neurodevelopmental disorder GRIN2B. They are currently looking into loading iPads and computers with telehealth capabilities and tools to help families connect with one another and continue their therapies at home.

Pushing Forward

Along with providing families with the technology to continue these routines, Sarah Chamberlin of National PKU News addresses the financial strain that at home care puts on families who are unable to work, or have reduced hours due to COVID-19. Sarah says that the grant will allow many families to be able to use the funds usually reserved for medical foods to pay other expenses.

“When COVID hit, we did a survey of our community asking what problems they were facing. Of the 280 responses, 25% of people said they had either lost medical nutrition coverage or were at risk of losing it due to COVID-19-related job loss or cutbacks.” – Sarah Chamberlin, National PKU News

Families from the non-profit Factor-H benefit from the Continuity of Care RARE Patient Impact Grant

“The role of foundations is really essential in investing in organizations and getting that stamp of approval for other funders to match or contribute more,” says Sarah. The importance of collecting data and leveraging community victories is what Sarah believes to be the most effective way to get necessary funding for rare disease communities.

At Fighting for Kaiden, Kris DeFazio hopes to tap into this by expanding the organization’s global reach and creating a space for spinal muscular atrophy (SMA) patients to store all of their patient data in one place. With Backpack Health, she explains,“this data is free and accessible to all researchers, and is a great way to get real-life data while helping patients to store all of their medical information in one place.”

As resources and research efforts continue to stream into finding a treatment for COVID-19,  more questions have begun to arise from the rare disease community. When will our research and clinical trials resume? How will the expedited testing and research we have seen made possible during COVID-19 affect drug development and treatment for rare diseases?

While right now we can only speculate at these answers, Liz believes we have a reason to be optimistic. “As a rare disease patient, we know what it’s like to have our world turned upside down. Something good that comes from this is that it forces us to think of some new and different ideas, and I’m so grateful for that.”

You can learn more about the recipients of the Continuity of Care RARE Patient Impact Grant, apply for a grant, or donate to help support the fund today  through our website. If you need tools to help you navigate the current pandemic, please visit our COVID-19 resource page.

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