University of Pittsburgh School of Medicine researchers carrying out a small pilot clinical trial demonstrating that a drug-free, minimally invasive intervention targets the root cause of progressive ...
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 ...
Children with spinal muscular atrophy (SMA), a rare autosomal recessive disease that results in progressive muscle weakness and atrophy, can suffer the concerning consequences of dysphagia, which ...
12hon MSN
Boy, 5, born with rare genetic disorder walks by himself after receiving world’s most expensive drug
Megan Willis' son, Edward, was given the gene therapy, Zolgensma, after being diagnosed with spinal muscular atrophy when he was 2 months old ...
UAE Moments on MSN
Abu Dhabi hospital first to administer pioneering gene therapy
Abu Dhabi has made medical history after Sheikh Khalifa Medical City (SKMC) became the first in the world to administer a ...
Fakeeh University Hospital is emerging as a global destination for advanced gene therapy, delivering highly specialized ...
Biogen said U.S. and European regulators will review its applications seeking approval of a higher-dose regimen of its Spinraza drug for the neuromuscular disease spinal muscular atrophy. The ...
Nicole Moore had contemplated getting her driver’s license before, but the progressive nature of spinal muscular atrophy, a neuromuscular disease she was diagnosed with at about 15 months old, held ...
Read before the Section for Clinical Medicine, Pathology, and Hygiene of the Suffolk District Medical Society, June 9, 1885.
A person may inherit the genetic changes responsible for muscular dystrophy. These genetic changes can also occur due to spontaneous genetic mutations. In either case, the disease is not preventable.
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