Living with a Rare Disease Worldwide. Volume 3

“Advances in artificial intelligence and biotechnology have spurred excitement in rare disease communities in the past year. The emergence of a revolutionary treatment for sickle cell disease, using the innovative gene-editing therapy CRISPR, signaled the possibility of additional advances that could help the more than 300 million people worldwide living with complex, mystifying and devastating illnesses.

Yet this progress comes in stark contrast to the reality that many rare disease patients worldwide face: a lengthy quest to get a correct diagnosis followed by the excruciating challenge of finding and affording treatment.

This disparity was at the center of the National Press Foundation’s 2023 “Covering Rare Disease” journalism fellowship, held online November 13-17 thanks to the generous sponsorship of Fondation Ipsen.”
Anne Godlasky (President, National Press Foundation, Washington D.C.)
Rachel Jones (Director of Journalism Initiatives, National Press Foundation)

“One of the great challenges faced by people living with rare diseases is that the public knows so little of their struggles, challenges, and the need for unrelenting resilience in a world that doesn’t understand. Coupled to the 300 million people living with rare diseases are hundreds of millions of caregivers who sacrifice time, work and money in the name of love. Working with the National Press Foundation, top world journalists highlight in these writings the plights of patients living with rare diseases and those who care for them. The stories are informative, dramatic, and heart-rending. I thank most the patients living with rare diseases and the people who love them for sharing their stories with these gifted journalists.”
James A. Levine (MD, PhD, Professor, President, Fondation Ipsen, Paris)

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