Newborn Screening Treatment Series: Part 1: Emerging Therapies and Access Strategies

The APHL New Disorders Subcommittee hosted a three-part webinar series that explored the latest advancements in treatments for both legacy and newly added newborn screening (NBS) disorders, and the critical challenges of access. Designed for NBS program staff, clinicians, policymakers, and partners, this series covered innovative therapies for NBS disorder, strategies for managing new conditions with limited treatment sites, and collaborative solutions to improve access. 

The first webinar explored new treatments for legacy NBS conditions with an emphasis on inborn errors of metabolism and cystic fibrosis (CF). Speakers highlighted how these new therapies are reshaping screening results, improving outcomes, and influencing long-term care strategies. Speakers also addressed access and availability challenges associated with these new interventions.