NEWS: First Duchenne Muscular Dystrophy Patient Treated in Follistatin Gene Therapy Trial

Cleveland and Columbus, OH, April 9, 2015—Milo Biotechnology announced the first Duchenne muscular dystrophy (DMD) patient was treated with follistatin gene therapy at Nationwide Children’s Hospital. The therapy, delivered by intramuscular injection, is designed to maintain or restore muscle function in boys affected with DMD. This six patient clinical trial follows from a trial of the therapy in Becker muscular dystrophy patients that demonstrated initial safety and efficacy. Results of that study were published in the January 2015 issue of the Journal Molecular Therapy.

The trial is being led by Dr. Jerry Mendell at Nationwide Children's Hospital. According to Mendell, "This is the first gene therapy clinical trial to demonstrate functional improvement in any form of muscular dystrophy, and a major advance for those suffering with muscle disease." The DMD trial is being funded by the Duchenne Alliance, which coordinated funding from 15 disease foundations from around the world.

The therapy, developed at Nationwide Children's Hospital by Dr. Mendell and Dr. Brian Kaspar, is based on adeno-associated virus delivery of follistatin 344 to increase muscle strength and prevent muscle wasting and fibrosis. Because follistatin’s mechanism of action is not mutation specific, it could potentially help other forms of muscular dystrophy. Another clinical trial in patients with inclusion body myositis is also being conducted at Nationwide Children’s Hospital. “We are excited about the broad potential for this therapy and are working with foundations and patient advocates to launch additional studies,” said Milo CEO Al Hawkins.

About Milo Biotechnology

Milo Biotechnology is a clinical stage startup company developing therapies to strengthen muscle and improve the lives of patients with neuromuscular diseases. Its lead program, AAV1-FS344, leads to the local expression of follistatin, a potent TGFβ ligand inhibitor inhibitor. Milo’s core technology was developed at and is exclusively licensed from Nationwide Children’s Hospital in Columbus, Ohio. The company is based in Cleveland, Ohio, and has received funding from JumpStart Inc., the Cuyahoga County North Coast Opportunities Fund, and from NIAMS Rare Disease SBIR program. Visit for more information.

About Nationwide Children’s Hospital

Ranked 7th of only 10 children's hospitals on U.S. News & World Report’s 2014-15 “America’s Best Children’s Hospitals Honor Roll” and among the Top 10 on Parents magazine’s 2013 “Best Children’s Hospitals” list, Nationwide Children’s Hospital is one of the nation’s largest not-for-profit freestanding pediatric healthcare networks providing care for infants, children, and adolescents, as well as adult patients with congenital disease. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s faculty train the next generation of pediatricians, scientists, and pediatric specialists. The Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded freestanding pediatric research facilities in the U.S., supporting basic, clinical, translational, and health services research at Nationwide Children’s. The Research Institute encompasses three research facilities totaling 525,000 square feet dedicated to research. More information is available

About Duchenne Alliance

The Duchenne Alliance is a group of independent non-profit organizations dedicated to defeating Duchenne Muscular Dystrophy. The Duchenne Alliance works to promote better collaboration among the many non-profit organizations around the world committed to conquering Duchenne and improving the lives of those living with this progressive and debilitating muscle wasting condition. The Duchenne Alliance member foundations collaborate to co-identify, co-review, and co-fund the most promising biomedical research. The Duchenne Alliance Research Fund was established to streamline their biomedical research programs. Please visit to follow and support our progress.


Al Hawkins



#pressrelease #MiloBiotechnology #NationwideChildrensHospital #genetherapy #DuchenneAlliance #follistatin #clinicaltrial #Duchennemusculardystrophy #DMD #Duchenne #Milo

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