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

Dennis P. Scanlon, PhD; Emma Ciafaloni, MD; Maria Lopes, MD, MS; Kevin U. Stephens, Sr, JD, MD; and Mary Schroth, MD, FAAP, FCCP provide an overview of the disease burden in spinal muscular atrophy and give insight into clinical and payor perspectives on screening and diagnosis, current treatment options, and new disease-modifying therapies in SMA.
EP 1: An Overview of the Epidemiology of SMA

April 20th 2021

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

EP 2: Clinical Burden of Spinal Muscular Atrophy

April 22nd 2021

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.

EP 3: A Multidisciplinary Approach to Diagnosis and Treatment

April 27th 2021

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.

EP 4: Screening and Diagnosis of SMA in Children and Adults

April 29th 2021

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.

EP 5: Challenges with Payor Systems for the Diagnosis of SMA

May 4th 2021

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.

EP 6: Adapting to Rapid Advancement in Treatments in SMA

May 6th 2021

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.