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A cryptic pathogenic NDUFV1 variant identified by RNA-seq in a patient with normal complex I activity in muscle and transient magnetic resonance imaging changes

A blend can be harder to verify than a single peptide, so the certificate of analysis matters even more

These cholesterol reductions brought levels comparable to those seen in lean, healthy control mice, suggesting comprehensive metabolic normalization

Sadri responded to FDA's Form 483 to explain the discrepancies, including delayed activation of the clinical trial site and shortening of the enrollment period, and said he would update the standard operating procedures