EGL Genetics Blog

PPMD Awards Gene Therapy Grant

Posted by Danyella Davis on Jan 25, 2017 3:00:00 PM

In an effort to further accelerate research within the Duchenne community, Parent Project Muscular Dystrophy (PPMD) awarded Dr. Jerry Mendell,  Dr. Louise Rodino-Klapac and Nationwide Children's Hospital with a $2.2 million grant.

"We are entering a new chapter in gene transfer therapeutics and while there are still questions to answer, the faster we move to answer these questions, the faster we know if a strategy will work," said Abby Bronson, SVP, Research Strategy, PPMD.  

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Topics: muscular dystrophy, Duchenne, neuromuscular disorders, Parent Project Muscular Dystrophy, Nationwide Children's Hospital, DMD, PPMD, gene therapy, clinical study, grant, Jerry Mendell

EGL is a Proud Supporter of Decode Duchenne, Jain Foundation, and the LGMD Consortium

Posted by Antonio Shaw on Dec 10, 2015 11:00:00 AM

Muscular dystrophy (MD) is a group of musculoskeletal diseases that cause progressive weakness, loss of muscle mass, and impaired locomotion. In muscular dystrophy, abnormal genetic variations impede the production of proteins necessary for healthy muscles. There are various types of MD, and the clinical presentations of the most common variety begin in childhood, primarily in males. However, other types of MD don't appear until adulthood.

With many of the neuromuscular disorders overlapping in their clinical and/or pathological phenotypes, molecular testing can be necessary to pinpoint the precise disorder a patient has.

EGL has partnered with three organizations, Parent Project Muscular Dystrophy, the Jain Foundation, and LGMD consortium to help spread MD awareness, provide assistance to individuals and families affected by MD, and find a cure.

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Topics: neurologic conditions, muscular dystrophy, genetic testing, dystrophy



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