Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA!

ABSTRACT:

Background/aims

Recruitment and retention of research participants are challenging and critical components of successful clinical trials and natural history studies. Infants with spinal muscular atrophy (SMA) have been a particularly challenging population to study due to their fragile and complex medical issues, poor prognosis and, until 2016, a lack of effective therapies. Recruitment of healthy infants into clinical trials and natural history studies is also challenging and sometimes assumed to not be feasible.

Methods

In 2011, our group initiated a two-year, longitudinal natural history study of infants with SMA and healthy infant controls to provide data to assist in the analysis and interpretation of planned clinical trials in infants with SMA. The recruitment goal was

SUBMITTER: Bartlett A 

PROVIDER: S-EPMC6072892 | biostudies-literature | 2018 Sep

REPOSITORIES: biostudies-literature