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What are the challenges when translating rare disease research into clinical development?

thumbnail - What are the challenges when translating rare disease research into clinical development?

6000-8000 rare diseases. A very small percentage of orphan drugs to treat. There is still so long to go and so much research to be done in the..

How to avoid late stage attrition rates when developing #orphandrugs

thumbnail - How to avoid late stage attrition rates when developing #orphandrugs

Innovation of orphan drugs to treat rare diseases is essential as the available treatment:rare disease ratio remains against the patients in..

Common mistakes to avoid during late stage #orphandrug development

thumbnail - Common mistakes to avoid during late stage #orphandrug development

Orphan drug development suffers from high late stage attrition rates, a challenge stifling all of pharmaceutical innovation. Experience goes..

What are the benefits of adaptive clinical trial design for your #orphandrug? #rarediseases

thumbnail - What are the benefits of adaptive clinical trial design for your #orphandrug? #rarediseases

Adaptive clinical trial design is increasingly becoming the buzzword for successful clinical development of your orphan drug. We surveyed and..

What are the major challenges for #orphandrug clinical trial design? #raredisease

thumbnail - What are the major challenges for #orphandrug clinical trial design? #raredisease

Orphan drug development to treat rare diseases has be proposed as an indication of pharmaceutical innovation in Europe. Moreover, successfully..

Rare disease patient groups – Saving Case at World Orphan Drug Congress USA

thumbnail - Rare disease patient groups – Saving Case at World Orphan Drug Congress USA

Melissa Hogan of Saving Case kindly provided this guest blog post. Melissa attended this year’s World Orphan Drug Congress USA, and presented..

Takeaways from the World Orphan Drug Congress USA – by Siren Interactive

thumbnail - Takeaways from the World Orphan Drug Congress USA – by Siren Interactive

Siren Interactive kindly provided this guest blog post from their blog, SirenSong. It was written by Eileen O’Brien, Director of Search &..

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