Provider and Caregiver Connection™: Living with Spinal Muscular Atrophy (SMA): Day to Day Patient Care

Provider and Caregiver Connection™: Living with Spinal Muscular Atrophy (SMA): Day to Day Patient Care is organized by Physicians' Education Resource, LLC (PER).

Release Date: July 31, 2019
Expiration Date: July 31, 2020

Target Audience:
This activity is intended for community pediatricians and neurologists, as well as physician assistants, nurse practitioners, primary care physicians, and nurses, involved in the diagnosis, treatment, and management of patients with SMA. This activity will also provide education to a broad multidisciplinary team, who are also invited to participate.

Accreditation/Credit Designation:
Physicians' Education Resource®, LLC, designates this enduring material for a maximum of 1.5 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Physicians’ Education Resource®, LLC, is approved by the California Board of Registered Nursing, Provider #16669, for 1.5 Contact Hours.

Activity Overview:
Spinal muscular atrophy (SMA) is a genetic disorder of the spinal motor neuron, with the majority of cases (95%) due to a mutation or deficiency in the SMN1 (survival motor neuron) gene on chromosome 5q13. This type of SMA occurs in 1 out of 11,000 births and is the primary genetic cause of death in infants. Spinal muscular atrophy is classified into 4 phenotypic groups based on age of onset and motor function; the earlier the age of onset, the greater the impact on motor function. Symptoms primarily involve the voluntary muscles, with those most affected closer to the central portion of the body, such as the shoulders, hips, thighs, and upper back. Complications can occur in which breathing and swallowing functionality is affected. Currently, there is no cure for SMA. Clinical management is primarily supportive care led by a pediatrician or neurologist using a vast array of healthcare disciplines, including physical therapists, nurses, rehabilitation specialists, speech and occupational therapists, pulmonologists, orthopedists, and nutritionists. Early diagnosis is imperative. Initiating care early in the course of disease may help to slow progression and loss of function.

This educational activity begins with a mother and caregiver’s story of her 2 daughters with different forms of SMA—from diagnosis and enrollment in clinical trials to an ongoing journey of challenges and successes. Two expert faculty then build on this caregiver perspective to discuss the pathophysiology and genetics involved in SMA, and how these translated into new treatments that can offer improved quality of life and hope for the future. Faculty conclude with strategies for multidisciplinary collaboration to optimize care of patients with SMA.

Learning Objectives:
Upon successful completion of this educational activity, you should be better prepared to:
• Recognize therapeutic targets in SMA and the mechanisms of action of the various pharmacologic agents
• Identify the clinical signs of SMA and integrate diagnostic testing along with the pathophysiology to better understand the disease and to initiate patient management
• Utilize knowledge of the adverse reactions and toxicities of SMA therapeutic options
• Apply patient perspectives to clinical practice, facilitating informed decision-making.