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 ...
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British boy walks after world's costliest gene therapy
A 5-year-old British boy suffering from the rare disease spinal muscular atrophy (SMA) has miraculously begun to walk four years after receiving the "world’s most expensive" gene therapy. On the 31st, ...
Megan Willis' son, Edward, was given the gene therapy, Zolgensma, after being diagnosed with spinal muscular atrophy when he ...
Medcare Women & Children Hospital has successfully administered a pioneering intrathecal gene therapy for Spinal Muscular ...
Muscles play a critical role in life. Skeletal muscle mass alone accounts for up to 40 percent of our body mass. Muscles turn chemical energy into mechanical energy and generate the power with which ...
Spinal muscular atrophy affects all the body's muscles. For a long time, it was considered a disease caused solely by the loss of nerve cells in the spinal cord. Now, a research team at Leipzig ...
Spinal muscular atrophy (SMA) and spina bifida are distinct conditions that affect the nerves that allow for voluntary motor control. Share on Pinterest press coverage photography/Getty Images SMA is ...
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 ...
Spinal muscular atrophy (SMA) is a genetic disorder caused by a homozygous recessive mutation in a gene on chromosome 5 (5q13), called the survival motor neuron 1 (SMN1) gene. It is due to deletion ...
They are sisters, born four years apart, both with the same cruel muscle-wasting disease that claims many sufferers in childhood. Yet their lives, and that of thousands of others like them, are being ...
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