pharmaphorum February 20, 2025
Around 75% of rare diseases are diagnosed in childhood, with most before the age of two. With 30% of rare disease patients dying before the age of five, the need for different treatment methods for children is clear.
In a new pharmaphorum podcast, web editor Nicole Raleigh speaks with Dr Helen Thackray, chief R&D officer at biotech company BioCryst, as well as an alumnus of the Children’s National Medical Center.
The conversation touches upon incentivisation for manufacturers to develop drugs to treat rare diseases, addressing age-appropriate formulations and recognising the...
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