IntroductionDuchenne muscular dystrophy, also known as DMD, is a serious hereditary genetic disease with no cure, which becomes evident in young men aged 1 at 6 years old. This disorder results from a frame-shifting mutation in the DMD gene, the third largest gene in the body, responsible for a muscle protein called dystrophin. This complex protein is vital for the function, structure and protection of muscles and muscle fibers. Mutation of this gene causes damage to skeletal and cardiac muscles as they repeatedly contract and relax. As this damage continues, muscle cells weaken and die, wasting away the muscle and causing heart problems at a very young age. Say no to plagiarism. Get a tailor-made essay on "Why violent video games should not be banned"? Get the original essay DMD is evident in about 1 in 3,500 boys in early childhood, prompting scientists, doctors and companies pharmaceutical companies around the world to collaborate and develop their lives. change the remedy for these boys. Through financial assistance and sharing of relevant data with international conventions, recommendations, innovative technologies as well as relevant research, companies and doctors such as "Sarepta", "Solid Biosciences" and Dr. Jerry Mendell have was able to conduct three individual studies. trials using gene therapy. In each trial, forms of microdystrophin were administered to patients through vectors, showing signs of hope for finding a cure for Duchenne muscular dystrophy. The SHE concept explored in this report is “Communication and Collaboration” and in particular the role this concept has played in the application of this revolutionary therapeutic technique. The diagnosis of DMD is made by examining a patient's weakness, developmental delay, increased serum creatine kinase (an enzyme released by damaged muscles), and confirmation by genetic testing and muscle biopsies. Current treatment of the disease with corticosteroids, bisphosphonates, and other medications aims to slow the onset of cardiomyopathy, prolong walking, and prevent loss of bone density. Exondys 51 is the only drug approved in the US and UK to directly treat DMD, as it is "designed to bypass the DMD gene mutation", but only works for a small number of people with a specific mutation . Thanks to advances in gene transfer technology, theories communicated by scientific communities around the world can finally be put into practice, with gene therapy becoming a possibility for people with Duchenne muscular dystrophy. Gene therapy aims to treat genetic diseases via correction of mutated genes at a molecular level in a clinical setting. This manipulation and replacement of the DMD gene is carried out using an adeno-associated virus (AAV) carrying a micro or mini dystrophin. As dystrophin is the third largest gene in the body, it initially seemed too big to be carried by a vector as it is three times larger than can fit in the vector, but scientist Jeff Chamberlain developed a micro- dystrophin and a mini-dystrophin. design that has 17 to 48 exons remaining. The development and results of micro-dystrophin through the collaboration of scientists have allowed international pharmaceutical companies such as Solid Biosciences, Sarepta, Pfizer and doctors to undergo clinical testingrecruiting patients to test the new treatment. In these trials, the viruses associated with adenoadenoses were stripped of their genes responsible for the disease and replaced with micro-dystrophin which will be injected into the patient's arm. The virus then moves into the cell membrane and transfers the genetic information ready for dystrophin protein synthesis. Risks associated with this therapy include unwanted attack by the immune system against the vectors, damage to healthy cells, and more serious infection caused by the vectors reverting to their original form. The Sarepta Therapeutics clinical trial was conducted at Nationwide Children's Hospital in Columbus by Dr. Jerry Mendell and in collaboration with the non-profit organization "Parent Project Muscular Project", this trial received a grant of 2.2 million dollars to finance the process. This funding contributed to the manufacturing of the vectors and costs for study participants who received the new treatment. In this trial, twelve patients from two cohorts aged 3 months to 3 years and 4 to years received a dose of micro-dystrophin designed for 60 to 70% of the DMD population. This study found that after 90 days, there was an increase in the amount of microdystrophin in the muscles of all patients, a significant decrease in the enzyme creatine kinase (CK), and no serious side effects. All results from this clinical trial show a positive outlook on the developments in gene therapy as a remedy for Duchenne muscle therapy. These results have been verified and reviewed by the Food and Drug Administration in the United States and the European Medicines Agency in the United Kingdom and, upon release, allow further investigation into the treatment internationally. The use of gene therapy to treat Duchenne muscle therapy has not only created hope for a cure for the genetic disease within global scientific communities, but it also has the potential to change the quality of life of young people boys around the world who struggle every day to complete basic activities. Mendell and his team at Nationwide Children's Hospital recommended further trials of this treatment before it was viable for all DMD patients, because participants who took part in the trial had little muscle atrophy . As soon as further investigations are completed and the treatment is approved by the FDA, EMA and TGA, communication between scientists, doctors and pharmaceutical companies internationally must take place for the implementation of the gene therapy in clinical practice. For families with a child with Duchenne muscular dystrophy, it can be distressing to see their child having difficulty getting up; walk, jump and run as their muscles weaken, but it is also possible that their heart muscles fail. This loss of ability to walk occurs between ages 7 and 13, and death is common among teenagers and early 20s. Any chance of their child moving normally is extremely important, as they may drop out of physical activities a young boy participates in, such as team sports, which are also important for social interactions. It is fair to say that for the average family income, treatment of this type is unfeasible even though it appears extremely beneficial to patients. This was noted after Sarepta and Mendell's trial: in January, when the trial began, the boys could barely climb a flight of stairs by holding on to the banister, but 90 days after treatment they were able to run, ride scooters and scooters.