Pharmaceutical Technology on MSN16d
Inborn errors of metabolism elicit unique challenges for therapy development
As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare diseases.
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Inborn Errors of Metabolism Drug Development Summit 2020
Inborn Errors of Metabolism Drug Development 2020 is dedicated to addressing these challenges to ensure that drug developers can achieve their full commercial potential. Access the full event ...
Nature6y
10 Differential Diagnosis in Patients with Developmental Delay: Focus on Inborn Errors of Metabolism
Developmental delay (DD) is common in paediatric practice and is present in 5-10% of children. Detailed evaluation can yield a definitive diagnosis in more than half of these cases and a ...