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Journal Review: Management of Spinal Muscular Atrophy in the Adult Population
Journal Review: Management of Spinal Muscular Atrophy in the Adult Population

Spinal muscular atrophy (SMA) is a group of neurodegenerative disorders resulting from the loss of spinal motor neurons. 95% of patients share a pathogenic mecha-nism of loss of survival motor neuron (SMN) 1 protein expression due to homozy-gous deletions or other mutations of the SMN1 gene, with the different phenotypes influenced by variable copy numbers of the SMN2 gene. Advances in supportive care, disease modifying treatment and novel gene therapies have led to an increase in the prevalence of SMA, with a third of SMA patients now represented by adults. Despite the growing number of adult patients, consensus on the management of SMA has focused primarily on the pediatric population. As the disease burden is vastly different in adult SMA, an approach to treatment must be tailored to their unique needs. This review will focus on the management of the adult SMA patient as they age and will discuss proper transition of care from a pediatric to adult cen-ter, including the need for continued monitoring for osteoporosis, scoliosis, malnu-trition, and declining mobility and functioning. As in the pediatric population, multidisciplinary care remains the best approach to the management of adult SMA. Novel and emerging therapies such as nusinersen and risdiplam provide hope for these patients, though these medications are of uncertain efficacy in this popula-tion and require additional study.

1)Understand the genetics and natural history of spinal muscular atrophy (SMA) and be able to apply these in diagnosis and in discussing prognosis ;
2) Be able to order and oversee optimal non-pharmacological management for adults with SMA;
3) Be able to understand, explain to patients, and order the most appropriate FDA-approved therapeutics for treating adults with SMA. 

The AANEM is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

The AANEM designates this enduring material for a maximum of 1 AMA PRA Category 1 Credits TM.  Physicians should claim only the credit commensurate with the extent of their participation in the activity. Credit expires 5/10/2025.

Dr. Rad is on a speaker's bureau for Biogen. Dr. Cai has nothing to dis-close. Dr. Weiss has consulted for UCB-Ra, Argenx, Alexion, Amylyx, MT Pharma, and Biogen. Conflicts of interest have been resolved according to ACCME guidelines.

Nassim Rad, MD; Haibi Cai, MD; Michael D. Weiss, MD
Availability: On-Demand
Expires on May 10, 2025
Cost: Member: $0.00
Non-Member: $25.00
Credit Offered:
1 CME Credit
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