Rare disorders and (potential) therapies

Today I had the chance to give a talk about "Rare disorders and (potential) therapies. 
Even though every single rare disease is rare on its own, many people are affected in the sum of all these diseases: In Germany, about four million people are affected. 
The technological development of recent years gives cause for optimism, as a rapid development in the clarification of the hereditary causes of these diseases can be observed. 
At the same time, the number of therapeutic approaches using genetic scissors or other modern methods from genetic research is increasing. 
I used selected examples to present causes and hopeful therapies for muscle disorder patients.