Reducing Disease Burden Through Early Diagnosis and Treatment of Spinal Muscular Atrophy (SMA)

An expert neurologist, Emma Ciafaloni, MD, provides an overview of the epidemiology of spinal muscular atrophy and discusses symptom burden, differential diagnosis, and prevalence.

A multidisciplinary panel of experts discusses the clinical burden of spinal muscular atrophy on patients, families, and caregivers and considers the importance of patient education and advocacy.

Key opinion leaders in SMA emphasize the importance of a multidisciplinary approach and centers of excellence in the early diagnosis and treatment of spinal muscular atrophy.

Experts in the management of SMA discuss the screening and diagnosis of infants, children, and adult patients and emphasize the importance of health care equity to avoid treatment delay.

Panelists provide insight into current challenges with payor models and early screening for SMA and discuss the importance of partnering with payors when rapid diagnosis and treatment are required.

A multidisciplinary panel of experts discusses the rapid pace of advancements in disease-modifying treatments and shares insights into how to work with payors to adapt and improve policies.

Emma Ciafaloni, MD and a panel of experts in SMA review the efficacy of nusinersen by discussing difference between clinical trial results and real-world clinical application.

Emma Ciafaloni, MD and Mary Schroth, MD, FAAP, FCCP discuss recent clinical trial designs and results for new disease-modifying treatments in SMA: onasemnogene and risdiplam.

Kevin U. Stephens, Sr., MD, JD and Maria Lopes, MD, MS provide insight into the factors that payors must consider when creating and implementing policies on treatment coverage in SMA.

Emma Ciafaloni, MD and Mary Schroth, MD, FAAP, FCCP consider the importance of shared decision making for patients and families when selecting an available DMT in SMA.

Experts in SMA provide closing thoughts on unmet needs in SMA including the importance of research into treatment combinations and sequencing as well as multidisciplinary care and healthcare equity.