Assessing the Future of Therapies in Development for
Limb Girdle Muscular Dystrophy
Podcast 2: Investigational therapies in LGMD
Post-Test/Evaluation

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A 60-year-old woman has been wheelchair dependent since she was 25 years old due to proximal weakness of

her lower extremities. She now presents with weakness of her upper extremities, and EMG shows a

myopathic pattern. She has significant muscle atrophy and weakness in most muscles, although some, like the

wrist, fingers, and toes, have been spared. Possible family history of muscular dystrophy, but not confirmed. A

recent muscle biopsy shows dystrophic findings, and normal staining for dystrophin, sarcoglycans, and

dystroglycans, but no staining for dysferlin. Genetic testing reveals heterozygous pathogenic variants in the

gene for dysferlin, DYSF. What type of LGMD does she have?

LGMDR2

LGMDR4

LGMDR1

LGMDD4

What are her treatment options? (Select all that apply)

Cardiac surveillance

Pulmonary surveillance

Physical therapy and rehabilitation

Genetic counselling

Clinical trials