RAO Network

PSC Partners Seeking a Cure

A group of 10 men and woman stand at a booth at the 2019 Liver Meeting in Boston
PSC Partners Seeking a Cure board members and staff attend the 2019 Liver Meeting in Boston, MA. Photo courtesy of PSC Partners Seeking a Cure.
PSC Partners Seeking a Cure board members and staff attend the 2019 Liver Meeting in Boston, MA. Photo courtesy of PSC Partners Seeking a Cure.

A collective voice to accelerate research

Patients and patient advocates are central to finding cures for rare diseases. They bring visibility to their disease, create strong and cohesive communities that help define unmet needs, and have an unrelenting passion to inspire research toward treatments and a cure. 

CEO Ricky Safer founded PSC Partners Seeking a Cure in 2005 after being diagnosed with primary sclerosing cholangitis (PSC). At the time, little information and support were available to PSC patients and their families. PSC is a disease of varying progression and a cluster of wide-ranging symptoms, making it difficult to point to a “typical” patient experience. Disease progression varies immensely, with some patients requiring a liver transplant within a year of diagnosis and others living 20 years or more without a transplant, though PSC can recur after transplant. No treatments exist for disease control or for slowing progression, and no cure exists.

Rachel Gomel has been an advocate for PSC with the organization for 13 years. She created a patient registry in collaboration with the National Institutes of Health (NIH), and with Ricky Safer, she was instrumental in initiating the PSC Forum, which aims to accelerate drug development for PSC. As an organization that knows the challenges that come with advocating for a rare disease, PSC Partners is most excited about being part of the Rare As One Network collaborative team and looks forward to learning from the experience of others in their respective rare disease areas and to expanding its perspective and possibilities.   

Patients and patient advocates have an unrelenting passion to inspire research toward treatments and a cure.

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