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There are ~7,000 RARE DISEASES.

400M PEOPLE are affected worldwide.

95% have NO TREATMENT (yet).  

Rethinking rare disease

We are an entrepreneurial pharmaceutical company working to drive change in rare diseases. 

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We're on a mission to get new drug treatments into the hands of patients. Quickly. Safely. Cost-effectively.

Developing new drug treatments 

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.  

Identifying undiagnosed patients

The journey to a rare disease diagnosis is an “odyssey” — taking an average of 6 years and marked by multiple misdiagnoses.

 

Our MagnetID  analytical service helps healthcare providers identify undiagnosed patients, drawing out rare disease cases like a magnet finding needles in a haystack.

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Harnessing our expertise

Some drug developers think rare disease treatments are unprofitable. And that patients are too hard to find. 
But they may not understand the 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. 

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Partner logo - Smartway.png
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Partner logo - UK Discovery Health.png
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Partner logo - MediWales.png
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