MEMPHIS, Tenn., Oct. 17, 2017 /PRNewswire/ -- Transnetyx, an automated genotyping company that serves biomedical researchers worldwide, today announced the premier of the documentary, "Brick in the Wall," as part of its storytelling collection, the Love Letter Series. The series chronicles the impactful stories of patients with rare disorders and the people who dedicate their lives to serving innovative research, aimed at finding a cure. "Brick in the Wall" follows the life and legacy of Joshua Frase, and his family's journey to find a cure for myotubular myopathy (MTM).
"We are grateful for the many families and patients who have shared their stories with us, especially the Frase family," said Bob Bean, President and co-founder of Transnetyx. "Without them, this testament to the powerful connection between research and laboratory care would not be possible. It is an honor to be a part of advancing the care and treatment of genetic disorders, particularly MTM."
MTM primarily affects muscles used for movement and occurs almost exclusively in males. People with this condition have weak muscles and decreased muscle tone that is usually evident at birth. It is characterized as one of the most severe childhood disorders, with lifelong morbidity, most requiring ventilator support, and early mortality is common. At the time of Joshua's diagnosis, there was no known cure for MTM. Faced with a grim prognosis, Joshua's family started a long journey and mission of finding a cure for this rare disorder. What they found along the way was hope in the form of a Labrador retriever, named Nibs, and the discovery that dogs can naturally be carriers and affected with MTM.
Successful gene transfer therapy in mice led researchers to a large animal model before a human trial could begin. Eager to expedite the next round of research that could potentially save her son, Joshua's mother, Alison, found Nibs, who was a female carrier of MTM. The inspirational short film, "Brick in the Wall" offers a unique perspective of how compassionate, dedicated animal care experts can not only change the trajectory of disease research—and the lives of patients and their families—but also serve as the guardian and friend of these lab animal heroes.
"Brick in the Wall" will premiere at the American Association for Laboratory Animal Science annual meeting in Austin, Texas, and can be viewed online here. The film distribution coincides with news that the first child has been treated with gene transfer therapy in a Phase 1/2 Clinical Trial for the treatment of X-linked myotubular myopathy.
"It has been an honor and a privilege to advocate for the last 22 years alongside these children and families, from diagnosis of this rare disorder to the recent commencement of clinical trials," said Alison Rockett Frase Joshua's mother and Founder and President of the Joshua Frase Foundation. "Sharing our story continues to fuel the fire of hope that one day soon there will be a cure—not just for MTM, but for many rare genetic disorders. We live in exciting times for research initiatives."
With more than 16 million samples processed, Transnetyx has served the research of more than 600 organizations—universities, hospitals and bioscience companies—worldwide, including 4,200 labs in the U.S., Europe and Australia. Through its automated model, Transnetyx continues to fulfill its mission by enabling scientists to accelerate research with fast, easy and accurate automated genotyping. This technology helps remove inefficiencies and human error in research so that life changing discoveries can reach patients quicker.
About Transnetyx Memphis-based Transnetyx is an automated genotyping company dedicated to serving research and saving time by removing redundant, yet necessary, tests conducted in genetics. Transnetyx has the technology and processes in place to provide faster, easier and more accurate results to biomedical researchers worldwide. Currently, the company has representatives in more than 10 major cities across the U.S., as well as in the UK, Europe and Australia. By optimizing resources, Transnetyx has paved the way for research to advance and deeper understandings to emerge. For more information, visit www.transnetyx.com. To find more stories like Joshua Frase's and the inspiring work of those who stand in the gap between research and the care for animals, visit www.loveletterseries.com.
About Joshua Frase Foundation The Joshua Frase Foundation (JFF) is a 501C3 nonprofit organization that was founded in 1996, one year after Paul and Alison's son was born with an ultra-rare and fatal neuromuscular disorder. Without hesitation, the foundation began funding cutting-edge research in the areas of regenerative medicine, gene therapy and genetics. JFF's mission is twofold: To find a cure or treatment for centronuclear and myotubular myopathies and to support families whose lives are affected by these disorders. Not only has JFF been able to fund groundbreaking science, pioneering into a curative treatment that has the potential to transform neuromuscular research; it has also built an international community of children, parents and researchers. For more information, visit www.joshuafrase.org.