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.
One-hour webinar, ‘Straightening the Growing Spine,” describes bracing and surgery options for children with a spinal curvature caused by a neuromuscular disorder
A one-hour, MDA-sponsored webinar features two physicians and the parent of a child with spinal muscular atrophy (SMA) who has undergone bracing and surgery for a spinal curvature, as well as questions and answers from listeners.
The National Institutes of Health has launched a biomarkers and natural history study in infants with type 1 spinal muscular atrophy
Update (March 28, 2013): This story was updated with a link to the ClinicalTrials.gov site for this study.
The pace of research can seem unreasonably slow; here are a few reasons why
John Porter from the National Institutes of Health likes to start talks by noting, “It’s a great time to be a mouse with a neuromuscular disease.” Exciting research results are regularly reported, where a treatment appears to cure one neuromuscular disease or another in a mouse — yet there are few treatments available today for people with any of these diseases, and only a few treatments in human...
Quest shares lessons from parents who have successfully helped their children with muscle diseases grow into self-reliant adults
When Vance Taylor was a boy, he didn’t know any adult he could look to and say, “There’s somebody like me.”
His mother, Morena Noyes, recalls the first time she took Vance and his sister Kathy — both of whom have limb-girdle muscular dystrophy — to MDA summer camp.
“We were still in the parking lot, in our Astro van,” Noyes says. “Vance looked, and then he turned to his sister and said, ‘Kathy,...
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Families, experts meet at BMD ConferenceThree trials study blood-vessel-dilating drugs in BMD, DMDResults of daily, weekly prednisone treatment about the same in DMD