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PCD is an autosomal recessive disorder of fatty acid oxidation caused by a dysfunctional organic carnitine (OCTN2) transporter
Multiple studies now demonstrate measurably better outcomes when the therapies are combined
To reduce discomfort or prevent potential adverse events: Start with the lower dose: Your doctor will likely begin with a small dose to minimize side effects
Administered as an oral rapid dissolve tablet (RDT)
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