Today is Duchenne Awareness Day #WDAD2021. Duchenne Muscular Dystrophy (DMD) is a devastating genetic disease characterized by progressive muscle degeneration, for which there is no cure. Most patients are diagnosed between the age of 2-5 years old and currently have a life expectancy of 30 years in the western world. DMD is caused by mutations in the dystrophin gene and affects all voluntary muscles in the body. NTrans’ iTOP DMD gene editing treatment will be used to preserve the critical arm function in DMD patients. Sustained upper extremity function is extremely important as it allows a patient to independently control his wheelchair, computer, phone and feed himself. It preserves a great level of independence and thereby significantly enhances the quality of life.
This week, NTrans has opened its new lab in Leiden (Biopartner 2). Please meet our team (f.l.t.r.): Sebastiaan, Niels, Marco, Silvana, and Marieke.
With this great team, NTrans aims to advance the development of its gene editing-based therapy for Duchenne Muscular Dystrophy (DMD).