It's estimated that only 5% of known rare diseases have an approved treatment.
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We acquire drug candidates that demonstrate, based on existing human safety and efficacy data, the potential for approval in rare disease indications where there's no current treatment.
Some drug developers think rare disease treatments are unprofitable. And that patients are too hard to find. But they may not understandthe rare diseases landscape like we do.
"New treatments for rare diseases are urgently needed – and I am excited to see that Ambrose is taking a different approach to the provision of new rare disease treatments.
It’s exactly what is needed to support patients."
Dr Jamie Duckers​
National Clinical Lead for Rare Disease, NHS Wales - UK
Collaborating is key
Discover a selection of our international network of rare disease organizations.