A Multidisciplinary Approach to Managing SMA

EP: 1.A 28-Year-Old Male With SMA

EP: 2.The Importance of Early Diagnosis of SMA

EP: 3.A 38-Year-Old Female with Spinal Muscular Atrophy

EP: 4.Diagnosis of Adult-Onset SMA

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EP: 5.A Multidisciplinary Approach to Managing SMA

EP: 6.The Safety and Efficacy of Nusinersen for SMA

EP: 7.Treatment Approach in SMA

EP: 8.Goals of Therapy and Treatment Selection in Adult-Onset SMA

EP: 9.Unmet Needs and the Future of SMA Care

EP: 10.Importance of Compliance and Multidisciplinary Management in SMA

EP: 11.Experience with Risdiplam and Unmet Needs in SMA

Arun S. Varadhachary, MD, PhD: The idea of a multidisciplinary team to care for patients receiving a diagnosis of a neurologic disorder has evolved over the past 15 years or so. The multidisciplinary team consists of clinicians from a variety of backgrounds who work together within the areas of their specialty to try to improve the health and functioning of patient. There is a growing body of evidence that a multidisciplinary team can help improve outcomes for patients with complex disorders, including spinal muscular atrophy (SMA). By working in a collaborative manner, individual parts of the patient's functioning can be addressed by experts in those areas. 

The multidisciplinary team may include the neurologist, who can help orchestrate a lot of care for the patient. However, other medical specialties are actively involved in the care of SMA patients, including pulmonologists to help improve respiratory function and orthopedic surgeons and physiatrists to help improve skeletal abnormalities that can develop as a consequence of weakness. Occasionally, we also work with our GI [gastrointestinal] colleagues to help optimize nutritional status in SMA patients. In addition to specific physician specialists, we also rely heavily on our colleagues in the physical therapy and occupational therapy realms. These professionals work regularly with patients to try to optimize function using stretching or strengthening exercises and other tools to help leverage their strength and compensate for areas of weakness. The goal of medication therapy is to try to address the underlying biology of the condition. That we now have treatment to address an underlying genetic disorder is a fantastic step forward. However, these medications are not perfect, and they need to work within the context of the overall care group. Thus, the goal of therapy is to try to optimize biology and work with more traditional strategies for care at the same time. The overall goal of therapy is, of course, to help the individual function at maximum potential. Since SMA affects multiple aspects of function, treating each one of those compromised aspects is necessary to maximize a patient's function.

Transcript Edited for Clarity