Newborn Screening Treatment Series: Part 2: Emergency 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 second webinar focused on disorders recently added to the NBS Recommended Uniform Screening Panel (RUSP), including metachromatic leukodystrophy (MLD) and Krabbe disease. Participants gained insight into the complexities of treating patients with these disorders and highlighted the challenges associated with accessing treatment.