Background: With the advent of antisense oligonucleotides (ASO) and gene therapies in spinal muscular atrophy (SMA), more patients are surviving into adulthood. Limited access to specialized adult neuromuscular and multidisciplinary care remains a significant barrier to effective pediatric-to-adult care transitions.
Objectives: To address this gap, we established the first regional adult neuromuscular clinic dedicated to SMA patients transitioning from pediatrics, bridging Connecticut Children’s Medical Center (CCMC) and University of Connecticut Health Center (UConn Health). We aimed to build institutional infrastructure for the integration of intrathecal ASO therapies and multidisciplinary services essential to SMA care, as well as design a patient-centered protocol to guide transitions from referral through initiation and maintenance of care.
Results: Four SMA patients have successfully transitioned their clinical care from pediatrics to UConn Health’s adult neuromuscular clinic, where they receive coordinated multidisciplinary evaluations. Referral, initiation, and monitoring protocols have been established and embedded within the clinic. While intrathecal ASO therapy continues being administered at CCMC, institutional approval has been secured to initiate therapy delivery through interventional radiology at UConn Health, with implementation anticipated in early 2026. Key barriers included limited resources to expand rehabilitation and allied health roles for multidisciplinary services and the complexity of coordinating across multiple hospital systems with distinct workflows (e.g., interventional radiology, pharmacy, electronic medical record).
Conclusions: Our experience underscores the importance of collaboration and early stakeholder engagement when establishing complex services within healthcare systems. Through this single center experience, we present a roadmap for other institutions to develop transitional care for SMA patients, highlighting essential steps and anticipated challenges. Future directions include expanding protocols to additional therapies and evaluating long-term outcomes and patient experience with patient-reported outcomes (PROs).