By the end of this activity, participants will be able to:

• Explain the role of cellular models in the preclinical stages of drug design and orphan drug development.
• Describe the process of biomarker development for clinical trials, including limitations and challenges to their development.
• Evaluate the impact of novel therapies, new screening technologies, patient advocacy, and new biomarker discoveries on driving recommendations for newborn screening.
• Assess novel clinical trial designs, recent advances and failures in gene therapy, ethical considerations, and the importance of registries and natural history studies, and integrate these insights integrate into research and clinical practice.
• Discuss the role and impact of Artificial intelligence in precision treatment of inborn metabolic disorders.