making muscle wasting history

Ever since genes were discoverer there has been talk of "personalised" medicine. The idea that we could adapt genes to treat specific diseases is a very powerful one. Exon skipping should make small molecule drugs look like a quick fix and medieval in comparison. However regulators have, quite rightly, been concerned that adapting genes on an individual basis would open patients to risks.

At the heart of this problem is the ambigious term "personalised".

In order to move forward we must distinguish make the case that personalised drugs means a personal service not just when the DNA is measured but also after the drug is given.

To most people it "personalised" means genomics. That is having drugs tailored to suit your body; for example some people respond better to asprin than paracetamol . Large clinical trials are undertaken on small molecule drugs because they will be given anonymously. So genomics simply means chosing between two drugs that have been through lengthy trials.

In order to move forward we must make the case that personalised drugs in DMD means non-anonymised (not tailored). This means a personal service not just when the DNA is measured but also after the drug is given. In DMD large clincial trials are not posssible so we need to work with regulators to de-anonymise the drug process so that exon skipping drugs are monitored and the regulators are assured.