In a Dec. 2, 2014, webcast for the Duchenne MD community, Sarepta Therapeutics explains plans, answers questions; the presentation is available on Sarepta's site
Cambridge, Mass.-based Sarepta Therapeutics, developer of eteplirsen and other "exon-skipping" drugs designed to treat Duchenne muscular dystrophy (DMD), provided overall reassurance about eteplirsen's development and some details of future plans to the DMD community in a Dec.
Isis Pharmaceuticals has opened a second phase 3 trial to test its "antisense" drug for spinal muscular atrophy in children ages 2 to 12
Update (Dec. 1, 2014): Isis has announced that its partner, Biogen Idec, plans to conduct a phase 2 trial of ISIS-SMNRx in up to 25 presymptomatic newborns genetically predisposed to develop SMA, as well as a phase 2 study of this drug in approximately 20 patients with infantile or childhood-onset SMA who do not meet the inclusion criteria for the current phase 3 studies. These two additional...
The investigational compound is designed to raise levels of the SMN protein, a lack of which is the underlying cause of spinal muscular atrophy (SMA)
PTC Therapeutics, a South Plainfield, N.J., biopharmaceutical company, says it will soon open a trial to test the safety and tolerability of its investigational drug RG7800 in adults and children with spinal muscular atrophy (SMA). The announcement was made in a Nov. 19, 2014, press release.
Four of six men with Becker muscular dystrophy increased their six-minute walking distance after injections of follistatin genes into the thigh muscles of both legs
Results from a trial involving injection of genes for the follistatin protein into the thigh muscles of both legs in six men with Becker muscular dystrophy (BMD) show the experimental treatment appears safe and and was associated with improvement in walking ability (distance walked in six minutes) in four of the six trial participants.
Israeli biotechnology company BioBlast is developing and testing Cabaletta, designed to counteract abnormal clumping of cellular proteins in oculopharnyngeal muscular dystrophy (OPMD)
Israeli biotechnology company BioBlast Pharma is developing an experimental drug designed to treat oculopharyngeal muscular dystrophy (OPMD) and is testing it in a phase 2-3 clinical trial at sites in Jerusalem and Montreal. If the U.S. Food and Drug Administration (FDA) approves the proposal, the company plans to open a trial site in the Los Angeles area of the U.S.
The first three infants in a gene therapy trial for spinal muscular atrophy have been treated; the trial continues, and additional trials are planned
Dallas-based biotechnology company AveXis, which is developing gene therapy for spinal muscular atrophy (SMA), announced in October that administration of its experimental gene transfer compound to the first three patients in a