Funding challenges in Rare Disease Research: How financial support can transform the Rare Disease Landscape

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Rare diseases may each affect only a small number of individuals, but collectively they impact up to 30 million Americans—highlighting a silent crisis in healthcare. Despite scientific progress, rare disease research remains chronically underfunded, resulting in delayed diagnoses, mismanagement, and a lack of effective treatments. This article explores the funding challenges that plague rare disease research and how strategic financial support—from government grants to public-private partnerships—can drive critical advancements. Transforming the rare disease landscape is not just about innovation; it's about giving millions a fair chance at timely diagnosis, effective care, and a better quality of life.

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Navigating Clinical Trials: A Guide for Rare Disease Patients

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Navigating Clinical Trials: A Guide for Rare Disease Patients

A condition is defined as a rare disease if it affects fewer than 200,000 individuals. Approximately 7,000 conditions meet this definition. In 2016, the United States Food and Drug Administration awarded $23 million dollars towards a four year period that supported research for 21 different rare diseases. However, most rare diseases lack disease-modifying therapies or effective medications for several reasons…

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CALL TO ACTION: Joaquin has diagnosis, now he needs a cure

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After six intense years of fighting against an unknown disease that burst into Joaquin's life at three years of age, and performing all the necessary exams to identify the disease and failing in each of them, Joaquin was diagnosed in March of 2017 through Rare Genomics’ philanthropic program iHope with Illumina.

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RG Success Story: Harrison Snow Starts Treatment for Rare Muscular Disease

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Since he was born, Harrison Snow had suffered from an undiagnosed muscular disease that led to difficulties speaking, swallowing, and breathing. He spent nearly every day in a state of fatigue and exhaustion. After four years of searching for a diagnosis, his family contacted the Rare Genomics Institute. 

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