Science, Maths & Technology
Rare diseases: low numbers, high impact
...public, on these diseases. What causes rare diseases? Most rare diseases (thought to be around 80%) have an inheritable genetic origin. These include HD and CF, mentioned above, and also a group of rare diseases collectively called Neuronal Ceroid Lipofuscinoses (NCLs). The NCLs include CLN3 disease (the juvenile onset form of the NCLs), which we are researching at The...