The administration of a protein called prolactin has been shown to slow weight loss, improve motor function and increase life span by approximately 70 percent in mice with a disease resembling a severe form of spinal muscular atrophy (SMA).
Prolactin is a hormone whose primary function is to promote milk production in women who are breastfeeding.
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.
Biotechnology company BrainStorm Cell Therapeutics has announced plans to collaborate with two American institutions to test its experimental stem cell technology in people with amyotrophic lateral sclerosis (ALS) in the United States. The institutions are Massachusetts General Hospital in Boston and the University of Massachusetts Medical School in Worcester, Mass.
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.