Rare Diseases

• Identify the criteria used to classify medical conditions as rare

• Summarize the social impact of living with uncommon illnesses

• Analyze geographic data regarding rare disease occurrences

• Explain how mutations drive most rare conditions

• Describe current tools used for identifying rare diseases

• Evaluate the role of external triggers in rare diseases

• Outline the challenges facing rare disease research studies

• Examine how cellular pathways fail during disease states

• Detail the consequences of misfolded proteins in patients

• Describe how metabolic errors trigger rare disease symptoms

• Analyze the process of creating drugs for rare diseases

• Assess the future role of gene editing therapies

• Evaluate how advocacy groups influence medical policy changes

• Propose strategies for managing rare disease patients effectively

• Synthesize current trends to predict future medical breakthroughs