Research for the treatment of rare diseases marked a decisive step forward.

The first human clinical trial for the cure of Huntington disease, a disorder originated from DNA-altering, will soon take place in London. The interesting thing about it, as reported in the article written by Laura Zuccato (Professor of Pharmacology at Milan University) for an important Italian newspaper, is the technique they are going to use, the Gene Silencing, which is the same employed by our researchers in the laboratory of the Dino Ferrari Centre for the treatment of CMT2A, in particular in the Scientific Project we have been funding for the last three years.
In our case, we are dealing with the silencing of Mitofusin 2 screwed-up gene and this stage of the trial made in laboratory with mouse model has paid off.
We are looking forward to have the test results of the London study, hoping that this clinical trial is going to open up new avenues for trials in the field of genic neuropathies due to Mitofusine 2.