Breakthrough Science
We relentlessly pursue bold science to translate genetic discoveries into new medicines that advance the future of human health.
Shola Fafunso is the U.S. country lead for the Skeletal Conditions business unit, responsible for driving the commercial efforts and aligning cross-functional teams to achieve business performance within this therapeutic area. But it’s the patients who serve as his North Star.
Reflecting on progress during her lifetime, Karlye discusses growing up with phenylketonuria, managing the condition amid a demanding career and starting her next chapter with high hopes for the future.
The holistic effort involving screening, data science and clinical evidence has the potential to help advance scientific understanding of hypochondroplasia and improve care.
We relentlessly pursue bold science to translate genetic discoveries into new medicines that advance the future of human health.
We listen to and learn from patient communities to collaborate on solutions with the potential to help improve care.
Our talented and dedicated people represent the best the biotech industry has to offer and have helped shape a culture focused on working together to deliver for the patients we serve.
Since our founding in 1997, we’ve applied our scientific expertise in understanding the underlying causes of genetic conditions to create transformative medicines, using a number of treatment modalities. Our approved medicines treat achondroplasia, severe hemophilia A and several rare inherited and lysosomal storage diseases. We’re also advancing an industry-leading pipeline of investigational medicines that are in various stages of clinical and preclinical development.
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Karlye is grateful that she's been able to pursue her passions, including sports and international travel, while growing up navigating PKU. Now as an adult, she is leaning into sharing her journey with the hope of helping others living with this rare metabolic condition.
Shola Fafunso is the U.S. country lead for the Skeletal Conditions business unit, responsible for driving the commercial efforts and aligning cross-functional teams to achieve business performance within this therapeutic area. But it's the patients who serve as his North Star.
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