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
Compounds designed to alter genetic instructions for dystrophin, increase production of utrophin, or prevent scar tissue formation in muscle are moving through the development pipeline
Drug development for Duchenne muscular dystrophy (DMD) is progressing on several fronts. Here are some updates as of early November 2014.
PTC is moving forward with ataluren
The American Academy of Neurology published recommendations for the care of people with limb-girdle muscular dystrophy and related disorders; MDA has endorsed the new guideline.
How the Recommendations Were Rated
Recommendations for diagnostic procedures or care were rated by the AAN panel according to the strength of the recommendation. The AAN uses the following rating scale: