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Robust clinical outcome measures for patients with juvenile onset GM1 gangliosidosis

Brianna Glase,Jean Johnston,Precilla D'Souza,Dee Adedipe,Cristan Farmer,Audrey Thurm,Cynthia Tifft

MOLECULAR GENETICS AND METABOLISM（2019）

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Key words
juvenile onset gm1 gangliosidosis,robust clinical outcome measures
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