• Small Molecule Therapy

    Definition: Small molecule therapy uses low molecular weight compounds (typically <900 Daltons) to treat diseases by modulating biological processes within cells. Key Features: Mechanisms of Action: Advantages: Applications: Examples: Challenges: FAQs:

  • Occupational Therapy

    Definition: Occupational therapy (OT) in rare genetic diseases involves interventions aimed at improving patients’ ability to perform daily activities and enhance their quality of life, despite limitations caused by their genetic condition. Key Concepts: Occupational Dimension: The impact of rare genetic diseases on a person’s ability to engage in meaningful…