Closer to patients in support of International Myotonic Dystrophy Awareness Day

Arthex Biotech is a spin-off of the University of Valencia, developing advanced treatments with RNA against genetic diseases. Our team has extensive experience in drug discovery and the study of the pathogenesis mechanisms of neuromuscular diseases. 

Arthex’s goal is to find effective treatments for unmet medical needs. The company is fully focused on developing treatment against Myotonic Dystrophy type 1 (DM1) based on antisense RNA. 

Thanks to our collaboration with MDF (USA), Euro-Dyma (Europe), ASEM, and BENE (Spain), we have successfully organized catch-up sessions to inform the patients about the development of our project. However, we need to open a more fluid communication channel with the protagonists of this story, the patients suffering from Myotonic Dystrophy and their closest relatives. So, we decided to conduct surveys among the patients who wanted to collaborate to find out their needs. 

We promoted these questionnaires on our social networks, the company website, LinkedIn, and groups formed by patients on Facebook. We tried to do everything possible to give it visibility, as we wanted to get closer to the more patients, the better. We still remember the joy when we read the first response from one of these patients. In fact, more than 96% of the participants in our survey were patients who suffer from Myotonic Dystrophy or their closest relatives. The patients in these surveys reflected that muscle symptoms such as myopathy, myotonia, and atrophy were the most limiting symptoms in their day-to-day lives. According to most patients, the muscular condition greatly impacts their daily activities, from something as simple as buttoning a shirt or opening a bottle to great mobility difficulties. All the responses reinforce our idea that the development of a therapy targeting the muscle for DM1 patients will have an important effect on their lives and has helped us a lot to encourage our work at Arthex.

Arthex Biotech is developing ATX-01, a new drug based on an antisense RNA therapy aimed at treating DM1. The main target of this new drug is the muscle, and its mechanism of action is based on the modification of gene expression by oligonucleotides that inhibit microRNA.