Breaking Down Barriers: Innovative Approaches for Streamlining Access to Rare Disease Therapies

Hope is often the driving force behind the relentless work of research teams dedicated to developing transformative therapies for the millions of individuals and families impacted by rare diseases. Through collaboration with manufacturers, patients, advocates, and clinicians, these researchers are advancing promising therapies into clinical trials and pioneering innovative tools and technologies that offer hope to the 300 million people worldwide living with a rare disease—95% of whom still lack effective treatment options.

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