Rare diseases

Oncology

Collaboration & personalisation in rare disease R&D, with AO...

For Rare Disease Day, in an AOP Health-sponsored podcast Melissa Fellner, VP of Global Therapeutic Areas, Commercial Operations, discussed their work.

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FDA maps new regulatory route for ultra-rare diseases

The FDA has sketched out its 'plausible mechanism' regulatory pathway for individualised therapies that treat ultra-rare diseases.

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FDA starts review of Regeneron's drug for rare disease FOP

Can Regeneron's drug for ultra-rare bone disease FOP pick up the baton from Ipsen's Sohonos, whose growth has stalled?

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NICE backs first disease-modifying drug for ARG1 deficiency

Immedica's Loargys is poised to become the first NHS therapy for ultra-rare genetic disorder ARG1 deficiency that can alter the course of the disease.

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