A drug’s mechanism of action, or MOA, refers to how a drug or other substance produces an effect in the body. Understanding a drug's mechanism of action (MOA) is crucial for a variety of reasons, including, but not limited to:
- Improved Understanding: knowing how a drug works can help individuals and families to feel more in control of their treatment and reduce anxiety about the unknown.
- Anticipating Side Effects: Understanding the MOA can help patients understand potential side effects and why they may occur, allowing for better management.
- Informed Decision-Making: Knowledge of the MOA can help patients make informed choices about their treatment options.
- Improved Communication: Patients who understand the MOA of their medications are better equipped to communicate their concerns and questions to their healthcare providers.
Cure SMA is committed to providing individuals living with spinal muscular atrophy (SMA) and their caregivers with resources for learning about treatments to aid in their decision making. To assist with this goal, we are pleased to begin updating our website with more information about drug MOAs beginning with gene therapy.
Gene therapy is broadly defined as the use of genetic material to treat disease. It aims to address the underlying genetic cause of disease. For example, in the case of SMA gene therapy can be used to introduce the missing SMN1 gene back to the cells of an individual with SMA.
To further explain gene therapy, we are pleased to present the following video focused on the MOA of gene therapy. While not drug specific, the video does discuss gene therapy in the context of SMA. Learn more about the basics of gene therapy and how it works here.
This video was created with support from Presenting Sponsors Novartis and Ultragenyx.
