Fast Five Quiz: Can You Recognize Kugelberg Welander Spinal Muscular Atrophy?

Stephen L. Nelson, Jr, MD, PhD


December 06, 2018

Despite ongoing research, there is no cure for Kugelberg Welander SMA; therefore, as with other types of SMA, treatment is mainly supportive, with a primary goal of slowing disease progression. Treatment of Kugelberg Welander SMA includes a rehabilitation program that includes physical therapy with preventive rehabilitation, occupational therapy to increase independence, speech therapy for patients who have dysphagia or who require diet modification, and other supportive services to maintain and preserve patient independence and assess future physical and emotional needs.

Regarding drug therapy, in 2016, the US Food and Drug Administration approved nusinersen, which has been shown to improve motor function and reduce mortality in patients with SMA. In addition, RG7916, an investigational drug similar to nusinersen, is currently undergoing clinical trials.

There are no immunotherapeutic agents available for Kugelberg Welander SMA.

Kugelberg Welander SMA is a progressive disease. While some case studies have reported exacerbation of muscle weakness in pregnant patients with Kugelberg Welander SMA, exacerbations are not common, and corticosteroid therapy is not appropriate. Beta-blockers and ACE inhibitors are also not indicated in the treatment of Kugelberg Welander SMA.

Read more about standard treatment and management of patients with Kugelberg Welander SMA here.


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