On July 17th, researchers at the University of Minnesota took a pivotal step towards finding a cure for Duchenne Muscular Dystrophy.
Myogenica, a University of Minnesota startup company, announced U.S. Food and Drug Administration approval for an Investigational New Drug (IND) application for MyoPAXon — an induced pluripotent stem cell-derived muscle stem cell product to regenerate skeletal muscle. A pending clinical trial would evaluate the safety, tolerability and engraftment of MyoPAXon in patients with Duchenne muscular dystrophy (DMD).
Dr. Peter Kang, a pediatric neuromuscular neurologist and director of The Greg Marzolf Jr. Muscular Dystrophy Center at the University of Minnesota Medical School, would lead the proposed early-stage clinical trial.
“We believe stem cells have enormous potential as a tool for muscle regeneration in muscular dystrophy and the timing is perfect to move toward a human clinical trial of this type of therapy in Duchenne muscular dystrophy,” said Kang.
If the proposed trial is successful, it has the potential to treat a range of muscular dystrophies, as well as other muscle degenerative conditions.
Patty Marzolf, President of The Greg Marzolf Jr. Foundation cheered this news, “This is a tremendous and very significant step in the GMJF’s quest for a cure for all living with MD. My heart is filled with gratitude, joy and new found hope for what is ahead.”
This development has been made possible by your steadfast support. Dr. Rita Perlingeiro previously expressed the impact of your commitment, “No words can really describe the impact of the Greg Marzolf Jr. Foundation funding to me and to our research over the years. The Greg Marzolf Jr. Foundation was the FIRST to fund this translational project. With seed funds from 2016-2018, we were able to produce enough data to be competitive for larger grants, including awards from the Department of Defense and Duchenne UK. In 2021, we could count again on the Greg Marzolf Jr. Foundation, when our funding was not sufficient to cover all the costs associated with the preclinical work required prior to IND-submission to the FDA.”– Dr. Rita Perlingeiro
Thank you again for your continued support in the Greg Marzolf Jr. Foundation, we are grateful for this community and the remarkable work of these researchers.
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