Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...
A study by Indiana University School of Medicine researchers sheds new light on the development and treatment of a rare form of muscular dystrophy. The study's findings were recently published in ...
-DMD preclinical data support candidate declaration for GB703, a potential DMD gene therapy designed to broaden mutation coverage and improve muscle delivery -SMA1 preclinical data support the ongoing ...
We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Motor function and other motor milestones were ...
At her eight-month pregnancy check-up, Lizmara Figueroa met with a new doctor. As he walked out the door, he made a parting comment. “He says, ‘Oh, just so you know, you’re a carrier for (spinal ...
Panelists discuss how exciting ongoing research efforts are expanding similar gene therapy technologies to other muscular dystrophies like FSHD and myotonic dystrophy, using strategies to knock down ...
New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of individuals with Types 2 or 3 spinal muscular atrophy (SMA) Late-breaking ...
Conserving energy is an essential component of living with Duchenne muscular dystrophy (DMD). Fatigue, both physical and mental, is extremely common with this genetic disease that causes progressive ...
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