Target group

SMA patients who would like to register should have a genetic test result that confirms a diagnosis of SMA, or have a genetic test arranged in the near future. Genetic confirmation of an SMA diagnosis is necessary so that any information provided by the registry is relevant to the patient's condition. Also, it is vital, when responding to researchers' enquiries into anonymised registry data, that each patient has a confirmed diagnosis of SMA. In addition, it helps the registry be sure that each patient has the appropriate genetic diagnosis for a particular clinical trial.

On the TREAT-NMD website, you can find a list of SMA registries in other countries where you might find the right registry with which to register if you are not resident in the UK or in Ireland. Information about DMD/BMD (Duchenne/Becker muscular dystrophy) registries and about registries for (other) rare inherited muscular disorders can also be found there.