Gene transformed his love of nature into intricately detailed wood carvings of wildlife. For many years he had a gallery named Waterfowl by Gene in Stockton and was active in the city’s Arts Council.
Complete and extremely encouraging findings from a phase 1b-2 trial of eteplirsen (AVI-4658), an exon skipping drug in development to treat a portion of the Duchenne muscular dystrophy (DMD) population, show the compound is safe and well-tolerated, and that it can significantly increase production of the needed dystrophin protein in recipients without eliciting an unwanted immune response.
A one-year, MDA-supported study comparing a weekend-only prednisone treatment schedule with a daily prednisone schedule in boys with Duchenne muscular dystrophy (DMD) has found that the two treatment regimens provide about the same benefits and have approximately the same side-effect profile.
When a medical emergency strikes — and the patient is a person with a neuromuscular disease— it’s not just getting to the emergency room quickly that’s critical. It’s also critical to ensure the ER staff understands the patient’s special needs caused by muscle disease.
Antisense oligonucleotides — also called antisense, oligos, or simply AONs— are pieces of genetic code that keep other genetic code from being processed. Designed to pair up with a particular sequence of DNA or RNA, AONs can change, block or destroy targeted genetic instructions in a variety of ways.
“I have a degree in biochemistry, and no experience in physiotherapy.” I kept reminding myself of this fact as Suse, a volunteer from Germany, and I first walked into the Duchenne Muscular Dystrophy (DMD) Centre for Boys in Bhaktapur, Nepal.
Belen Pappa, a graduate student in genetic counseling, is seeking participants for a survey-based study about psychosocial functioning in families in which a child has Duchenne muscular dystrophy (DMD).
The pattern of muscle loss in BMD usually begins with the hips and pelvic area, the thighs and the shoulders. To compensate for weakening muscles, the person may walk with a waddling gait, walk on his toes or stick out the abdomen.
The rate of muscle degeneration varies a great deal from one person to another. Some men require wheelchairs by their 30s or later, while some manage for many years with minor aids, such as canes.