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[DOI] [PubMed] [Google Scholar] 101.Jariwalla R., Lalezari J., Cenko D., Mansour S.E., Kumar A., Gangapurkar B., Nakamura D
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Ten subjects continued into an open-label extension, of whom eight reached 36 weeks, and at that point the investigators reported a change of +95.9 m on the 6-minute walk test (p = 0.024) and -2.1 points on the Barth Syndrome Symptom Assessment (p = 0.031), together with reported improvements in secondary endpoints including knee extensor strength, patient global impression of symptoms and some cardiac parameters (Reid Thompson et al., 2021)
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