The Bill is for all doctors and all patients, irrespective of the condition or disease they present with. However, rarer conditions are a particular issue and one with which the Bill is most likely to help.

Standard procedures are based on what is called ‘Big Data’ and evidence-based medicine – that is, they are designed around what is known to work. At least that is the theory. And it is fine when it comes to common conditions, such as diabetes and broken legs, where there are many patients and where theories have been well-tested and proved.

But standard procedures based on rarer conditions, where the data and evidence is patchy is a problem. If there isn’t an evidence base for a disease, it makes innovation difficult – because there are not the numbers of patients available to develop new treatments.

And it means, also that less research is carried out because no one wants to spend a millions of pounds researching drugs into rare diseases for which the market is small. Also, it is harder to recruit scientists to investigate rare diseases, for which funding is hard to come by.

Big diseases tend to carry the day. This relegates the rarer conditions into a scientific and medical backwater with doctors left able only to repeat their failures.

This is why we need to free doctors to innovate, safely, with and for patients.

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“In our case the risk of doing nothing is not nothing, the risk of doing nothing is fatal.

“Fatal every single time. You never survive this.

“What we are not willing to do is assume the risk of doing nothing.

“If a potential therapy shows promise of stabilisation or improvement over what would be expected without any treatment, and it shows safety, then patients and parents should be given a choice to try it with long-term studies taking place concurrently.

“Because, at the end of every discussion and assessment of a therapy, we must never lose sight of the reality that the risk of having Duchenne far outweighs the risk of most potential treatments.”

Alex Smith runs Harrisons Fund, a charity that funds research into Duchenne Muscular Dystrophy – a 100% fatal condition with no cure.

→WATCH: Alex speak at the House of Lords.