Duchenne muscular dystrophy: advances in therapeutics
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Xtalks Vitals is news, blogs, videos, quizzes and more! Duchenne muscular dystrophy is becoming a substantial target of rare disease drug development with pharmaceutical giant Pfizer joining smaller biotechs in the race to release more targeted therapies for the genetic disease. Through collaboration and ongoing dialogue with companies like Pfizer, we hope to succeed in finding therapies that could dramatically change the outcomes for those with [Duchenne muscular dystrophy]. The mini-dystrophin gene therapy was originally developed by Bamboo Therapeutics, which was acquired by Pfizer in The multi-center trial will involve about 12 boys between the ages of five and 12 who have been diagnosed with Duchenne muscular dystrophy but have retained the ability to walk.
The mini-dystrophin gene therapy PF will be infused intravenously, and the research team will assess its safety and tolerability in the patients. Prior to enrolling patients in the study, they will be tested for the presence of antibodies against the adeno-associated virus serotype 9 AAV9 capsid, which is used to introduce the gene therapy into cells, as well as an immune response to the dystrophin protein itself.
Since the gene therapy is designed to replace the absence of endogenous dystrophin, Pfizer will assess levels of dystrophin expression in the trial participants, along with whether the gene therapy is associated with changes in muscular strength and function. As an exon-skipping drug, Exondys 51 is only beneficial to patients with a mutation in the dystrophin gene on exon This trial will assess the safety of this approach to gene therapy and could provide valuable data demonstrating its potential impact to slow down or stop the progression of [Duchenne muscular dystrophy].
Pfizer anticipates that four clinical research sites across the US will be involved in the study and will continue to recruit patients in order to meet enrollment targets. Not a member yet? Sign up for our newsletters and receive webinars, vitals, and updates for your topic of choice. You have just entered the contest with submissions and point s spent.
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