There are many different types of research that can be done to improve our understanding of PHTS and the lives of patients. These include:
These capture information about what it is like to live with PHTS day to day and what patients see as the most pressing issues.
Also known as natural history studies. These provide an understanding of how PHTS progresses over an individual’s lifespan. This includes monitoring if and when symptoms show up, their impact on a person’s physical and mental wellbeing and how symptoms respond to treatment. Among other things, the data are used to calculate the risk of developing these symptoms among the wider population of people with PHTS.
If data about patients is collected in a consistent and rigorous way, statistical analyses can be done to look for trends and answer epidemiological questions such as who and how many people have PHTS. These analyses can also uncover similarities between patient groups, potentially uncovering vital information on the nature of the different syndromes related to PHTS.
This is the process of identifying and developing potential new drugs. Once scientists have a good understanding of the molecular biology that underpins the disease, large libraries of chemical compounds can be screened to see if they have any effect on it.
Chemicals that show promise in drug discovery screens are tested to explore their chemical and physical properties in the body – how safe or toxic they are, how quickly they are broken down and how they move around, for example. These tests are done on ‘disease models’, for example a collection of cells, or sometimes an animal such as a mouse, that have been engineered to show some of the characteristics of PHTS in humans.
Scientists also use disease models to test whether drugs that are already used to treat another condition might work on PHTS, and to refine their understanding of the disease.
Once preclinical research has confirmed that a potential new drug shows promise, trials are done to assess the safety, effectiveness and dosage in people with PHTS. Participants are given the drug and carefully monitored to see its effects.