Jerry Mendell

The first three infants in a gene therapy trial for spinal muscular atrophy have been treated; the trial continues, and additional trials are planned

posted on November 18, 2014 - 9:24am
Dallas-based biotechnology company AveXis, which is developing gene therapy for spinal muscular atrophy (SMA), announced in October that administration of its experimental gene transfer compound to the first three patients in a

A study to determine the natural history and outcome measures for clinical trials in Becker MD and sporadic (nongenetic) inclusion-body myositis is open in Ohio

posted on November 18, 2013 - 5:00am
A study to determine the best outcome measures — ways to evaluate the effects of a treatment — for use in clinical studies in Becker muscular dystrophy (BMD) and sporadic (nongenetic) inclusion-body myositis (sIBM) is underway at Nationwide Children's Hospital in Columbus, Ohio, under the supervision of neurologist Jerry Mendell working with physical therapists Linda Lowes and

Early data show follistatin gene transfer may be safe and effective in BMD and safe in IBM, in which efficacy has not yet been evaluated

posted on November 7, 2013 - 3:00pm
Preliminary results from a trial to test the safety of injecting follistatin genes into the thigh muscles of adults with Becker muscular dystrophy (BMD)or sporadic (nongenetic) inclusion-body myositis (sIBM) suggest that the experimental therapeutic approach is safe in both types of patients
posted on October 3, 2013 - 9:05am
Quest Vol. 20, No. 4
In kindergarten, Mo Gerhardt played tee ball and soccer, and while his hand-eye coordination was excellent, his strength wasn’t. Matthew (“Mo”) Gerhardt of Bath, Mich., will be 36 years old this fall.

Proposals for implementation of widespread screening of newborn babies for Pompe disease and Duchenne muscular dystrophy were delivered to a federal advisory committee

posted on March 20, 2013 - 12:07pm
Update July 22, 2013: This story was updated to reflect that, in April 2013, the Discretionary Advisory Committee on Heritable Disorders in Newborns and Children (DACHDNC) was chartered and now fulfills the functions previously undertaken by the Secretary's Advisory Commitee on Heritable Disorders in Newborns and Children (SACHDNC).

Longtime MDA research grantee Jerry Mendell discusses his group's development of a feasible strategy for screening newborn babies for Duchenne MD

posted on May 1, 2012 - 4:39pm
An April 2012 podcast from Nationwide Children's Hospital in Columbus, Ohio, explores the implications of a recently developed strategy for newborn screening for Duchenne muscular dystrophy (DMD). The podcast is part of a Nationwide Children's series called "This Month in Muscular Dystrophy."

Longtime MDA research grantee honored for work in LGMD gene therapy

posted on October 17, 2011 - 3:00am
A team headed by neurologist Jerry Mendell, a longtime MDA research grantee and director of the MDA Clinic at Nationwide Children's Hospital in Columbus, Ohio, has received the prestigious Annals of Neurology prize for an outstanding contribution to clinical neuroscience.

Gene therapy researcher and MDA grantee Jerry Mendell discusses autoimmunity to dystrophin protein in Duchenne muscular dystrophy

posted on January 13, 2011 - 3:57pm
A January 2011 podcast from Nationwide Children's Hospital in Columbus, Ohio, probes a subject that's been on the minds of many researchers, doctors and families: autoimmunity (self-immunity) in Duchenne muscular dystrophy (DMD).