Neurologist Katherine Mathews discusses cardiac, respiratory and other concerns for people with limb-girdle muscular dystrophy
Neurologist Katherine Mathews
Katherine Mathews, M.D., is a neurologist at the University of Iowa Hospitals & Clinics in Iowa City, where she co-directs the MDA clinic. She serves on MDA's Medical Advisory Committee. Below she discusses limb-girdle muscular dystrophy with MDA Medical and Science Editor Margaret Wahl.
A 29-year-old man with Duchenne MD has received a device to assist his heart's pumping ability
A left ventricular assist device (LVAD), which helps the heart pump blood throughout the body, was implanted in the chest of a young man with Duchenne muscular dystrophy (DMD) at Cincinnati Children's Hospital Medical Center in September. The patient is 29-year-old Jason Williams, of Peebles, Ohio.
A study of 11 long-term survivors of infantile-onset Pompe disease shows enzyme treatment can provide significant benefits, but deficits remain
A study of 11 children with infantile-onset Pompe disease (acid maltase deficiency) who started enzyme replacement therapy by the time they were 6 months old has shown the treatment can markedly improve the course of the disease, but that residual deficits persist.
A phase 2-3 clinical trial is testing whether an existing heart drug, eplerenone, can stop or slow heart damage in Duchenne muscular dystrophy
Eplerenone, a drug commonly used to prevent scarring after a heart attack, is being tested in a phase 2-3 clinical trial to determine whether it can stop or slow heart damage in people with Duchenne muscular dystrophy (DMD).
Eplerenone blocks scarring that over time can turn heart muscle into nonfunctioning fatty tissue.
The drug improved heart function and survival in mice with lamin A/C mutations, which cause lamin A/C-related Emery-Dreifuss MD and type 1B limb-girdle MD
Update (June 1, 2012): This story was updated to include the availability of a podcast on this topic.
Findings on the immune response to dystrophin in Duchenne and Becker MD, treatment of cardiomyopathy in DMD, and eteplirsen in DMD were presented
Update (June 1, 2012) — The brief Drugs help DMD-related cardiomyopathy has been updated to include a May 2012 podcast provided by Nationwide Children's Hospital's "This Month in Muscular Dystrophy."
Three recent MDA-supported studies investigate the nature of cardiac problems in Duchenne/Becker MD and propose possible treatment avenues
In both Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), deterioration of the heart muscle, a condition known as cardiomyopathy, is a major cause of disability and death.