Old dogs and new tricks – drug repurposing for rare diseases
In the same week as rare disease day and a summit on CAR-TCR looking at the ‘value’ assessment for incredible new gene and cell therapies...
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Patient intelligence and artificial intelligence combine to bring rare disease breakthroughs
As Rare Disease day approaches, it seems timely to reflect on some of what's new in rare disease.
A new scientific paper published...
Engaging with people and their stories of life with a Rare Disease
I have worked on multiple Rare Disease projects throughout my career in the Biopharmaceutical and Consulting industry but I am still...
Flipping the traditional drug development model on its head; Custom-designed CRISPR therapy is close to becoming a reality
Rich Horgan, the founder of the nonprofit Cure Rare Disease, is on a quest to develop a therapy for his brother Terry who was born with...
On the Transformative Potential of Advanced Therapeutics in Rare Disease
The burgeoning field of cell and gene therapies collectively referred to as Advanced Therapeutics (ADVTX), is at last beginning to...
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