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...
A 26-week study by AVI BioPharma finds that intravenous AVI4658 appears safe and beneficial to Duchenne MD patients with mutations in or around exon 51
The experimental drug AVI4658, in development by AVI BioPharma to treat Duchenne muscular dystrophy (DMD) caused by specific genetic mutations, was well tolerated and resulted in increased production of the needed dystrophin protein. Measured aspects of cardiac, pulmonary and skeletal muscle function remained stable.
Featured in this issue: Duchenne and Becker MD ** Facioscapulohumeral MD ** Friedreich's ataxia ** Limb-Girdle muscular dystrophy ** Myotonic dystrophy ** Spinal muscular atrophy
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
Phase 1 trial in boys with DMD shows 'robust' results
On Jan. 21, AVI BioPharma of Portland, Ore., announced its experimental compound AVI4658 for the treatment of Duchenne muscular dystrophy (DMD) yielded promising results in a phase 1 clinical trial in the United Kingdom.