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Identification of Tongue Involvement in Late-Onset Pompe Disease

CompletedN/A

Sponsor: Duke University

Conditions: Myopathy, Neuropathy, Glycogen Storage Disease Type II (Late-onset Pompe Disease)

Interventions: Observational study

Countries: United States

This purpose of this study is to determine if tongue strength and tongue ultrasound measurements differentiates patients with untreated late-onset Pompe Disease (LOPD) from patients with acquires/hereditary myopathies or neuropathies. It is hypothesized that abnormalities in tongue function and structure in patients with LOPD may be useful in discriminating this condition from others that have similar presentations.

Eligibility overview

Sex: ALL

Age: 12 Years to —

Healthy volunteers: No

Study type: OBSERVATIONAL

Eligibility criteria

Inclusion Criteria:

* age ≥ 12 years
* confirmed diagnosis of LOPD and naïve to enzyme-replacement therapy (ERT)
* acquired/hereditary myopathy (e.g., dermatomyositis, polymyositis, inclusion body myositis, limb-girdle muscular dystrophy, distal myopathy, myotonic muscular dystrophy, and other myopathy)
* neuropathy (e.g., peripheral neuropathy, cranial neuropathy, autonomic neuropathy, focal neuropathy)

Exclusion Criteria:

* current use, history within the past two years of use, or eligible but declined use of Lumizyme® enzyme replacement therapy (applicable to LOPD group)
* history of stroke, Parkinson's disease, oculopharyngeal muscular dystrophy, head and neck cancer or radiation treatment to head/neck, or other conditions that commonly affect lingual strength
* inability to follow directions for study participation

Locations (1)

Durham, North Carolina, United States