This subproject is one of many research subprojects utilizing theresources provided by a Center grant funded by NIH/NCRR. The subproject andinvestigator (PI) may have received primary funding from another NIH source,and thus could be represented in other CRISP entries. The institution listed isfor the Center, which is not necessarily the institution for the investigator.The primary objective is to assess the safety, tolerability, and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in SMA patients 2-17 years of age.
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