Few things are scarier in life than dealing with a sick child. But for some parents, that fear is compounded into a living nightmare when they are told their child has a fatal genetic disorder. That’s the terror faced by parents of boys diagnosed with Duchenne muscular dystrophy, or DMD.
This rare condition affects some 1 in 3,500 to 1 in 5,000 boys around the world. Patients with Duchenne are missing a protein called dystrophin that is critical to muscle function. Without it, their muscles gradually waste away, leading to loss of strength and mobility. Most DMD patients only make it into their 30s before passing away from respiratory or heart failure.
To date, there have been few good options for treating DMD. But in cases like this, where a disease affects a very small number of patients and there are no treatments available, the U.S. Food and Drug Administration leaves the door open to grant special and accelerated consideration for promising therapies.
Last fall, Cambridge-based Solid Biosciences stepped through that door when their investigational gene therapy for DMD was granted “Orphan Drug” designation by the FDA. The designation means that the therapy, currently called SGT-001, has met certain criteria for orphan status dictated by the FDA. It makes the treatment eligible for special incentives to assist with the development process, like tax credits to help with clinical testing.
With the orphan status in place—and with a similar designated granted by the European counterpart of the FDA—Solid Biosciences is moving ahead with a clinical trial for later this year. They’ve also opened a London office to facilitate working with the DMD community in Europe. And so far, that work is promising: preclinical work suggests that SGT-001 could help improve muscle function and strength and protect against future damage.
It’s early days yet for this potential treatment, and until the trial gets underway the story of SGT-001 and it’s impact on DMD has yet to be written. But maybe now, parents of kids with DMD can look forward to a story with much less horror and a lot more hope.
Read more about this exciting development here, then head over to Solid Biosciences keep up with the latest news.
