At Rare Disease Research, we aim to optimize the process of conducting clinical trials in rare disease conditions. To improve access to investigational therapies, increase opportunities for trial participation, patient recruitment and retention, we have an experienced team with a multidisciplinary approach to orchestrate clinical research trials in rare disease conditions. We closely collaborate with sponsors, CRO, and patient advocacy groups to improve awareness and recruitment for cutting edge clinical trials. Because rare diseases are not often considered a public health matter, we tirelessly advocate for newborn screenings. We want to improve health outcome for infants with rare genetics conditions who otherwise would have to endure diagnosis odyssey and delayed treatment. Our newborn screening project for Spinal Muscular Atrophy will begin in 2018.