Case Study – Pediatrics Study In Mucopolysaccharidosis Type III (MPSIIIA)

Discover how our scientists performed an open, non-controlled, parallel, ascending multiple-dose, multicentre study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of a chemical compound in paediatric patients with mucopolysaccharidosis type iii (MPSIIIA)

In paediatric studies, one of the biggest challenges is the permitted and available blood volume. Blood sampling from children has strict limits, and there are different guidelines from globally recognized bodies. In addition, sampling materials such as needles, IV-lines, and more need to be adjusted for children. Sample loss in these instances is near catastrophic for all the above reasons and accentuated in orphan indications.

Read how our scientists were able to realize this study.