Accelerating Clinical Studies in Rare Diseases
Rare diseases refer to those which have low occurrence but are complicated, mainly caused by genetic defects (such as spinal muscular atrophy and Fabry disease). In studies for rare diseases, to fulfill patients’ unmet needs has been advancing scientific breakthroughs and treatments. Each study needs an empathetic and professional team to handle the complexity and deal with the unpredicted issues. Kun Tuo is to provide clinical development services involving full lifecycles and settle the recruitment difficulty through the IQVIA global patient community network. The adaptive trial design will be adopted to cope with the challenges of small sample size and take every effort to benefit patients of rare diseases with breakthrough therapies.