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Spinal Muscular Atrophy SAPPHIRE SAPPHIRE was a randomized, double-blind, placebo-controlled Phase 3 clinical trial that evaluated the safety and efficacy of apitegromab in non-ambulatory patients with Types 2 and 3 SMA who are receiving current standard of care (either nusinersen or risdiplam)

Pen Dosage Chart Dosage & Protocols Variations Standard Research Protocol Dose: 5 10 mg daily (= 1530 clicks) Duration: 2 4 weeks Frequency: Daily Cycle Interval: 2-week pause Goal / Description: For baseline NAD+ and fat metabolism studies

1 Introduction Amino acids are organic compounds containing both amino (NH 2 ) and carboxyl (COOH) groups

Dart RC, Erdman AR, Olson KR, et al

The liver was dissected and divided into several sections for biochemical, molecular, and histological analyses