Assignment task:
Need help writing something good to this classmates post: Duchenne Muscular Dystrophy is an x-linked neuromuscular disease that causes a mutation in the patient's dystrophin gene. The disease is found predominantly in male patients. The effects of the disease can vary from weakness caused by degeneration of skeletal/cardiac muscles, trouble breathing, walking abnormalities, and heart conditions (specifically cardiomyopathy). Many patients walking ability are affected by 3-5 years old and wheelchair bound by 12 years old. The degeneration of muscles is caused by the lack/no dystrophin present in DMD patients' muscles, instead, fat/fibrotic tissues take place of healthy muscle fibers. See the image below of the difference between normal tissue and a DMD patients tissue: I chose this disease because not only am I doing my bonus project on this topic, but I am an undergraduate intern at a CRO where we are helping conduct clinical trials for patients that suffer from this neuromuscular disease. There are a few known investigational products that are helping patients who suffer from DMD, but the main game-changing therapy will be gene therapy because of this advancement in technology there may be a way for DMD patients to produce dystrophin themselves.