Complete results of a U.K. trial of an exon-skipping drug in boys with DMD show drug is safe, increases dystrophin production
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.
Clinical trials of two different exon-skipping compounds show encouraging results; Duchenne MD participants are being sought for new trials
sClinical trials that use compounds called antisense oligonucleotides to cause skipping of exon 51 of the dystrophin gene in individuals with Duchenne muscular dystrophy (DMD) are moving forward in the United States and elsewhere.
Exon skipping for DMD is a strategy that coaxes muscle fibers to ignore, or "skip," the genetic instructions for certain parts of the dystrophin gene so that functional...
Further analysis of data from a trial of the exon-skipping drug AVI4658 showed some participants had greatly increased dystrophin production
The biopharmaceutical company AVI BioPharma has announced additional encouraging results from its clinical trial of AVI4658, an experimental treatment for Duchenne muscular dystrophy (DMD).
The new results show that, at higher doses, AVI4658 can result in substantial production of the needed dystrophin protein in muscle fibers.
The company has not yet released results of any tests of muscle...
A trial of AVI4658, an experimental exon skipping construct for DMD, resulted in dystrophin production and appears safe
AVI4658, an experimental treatment for patients with Duchenne muscular dystrophy (DMD) caused by certain mutations in the gene for the muscle protein dystrophin, has shown promising results when delivered intravenously to 19 trial participants.
Early results show that when AVI4658 is delivered system-wide through the bloodstreams of boys with DMD, it’s safe and increases dystrophin production.
Interim results from a human clinical trial of the exon-skipping compound AVI4658 in boys with Duchenne muscular dystrophy (DMD) show that when the compound is delivered to the whole body via the bloodstream — rather than simply injected into a foot muscle as in a previous trial — it appears safe and leads to production of the missing muscle protein dystrophin.
The following article contains items about: Duchenne and limb-girdle muscular dystrophies
This article contains items about: Duchenne muscular dystrophy and spinal muscular atrophy