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Study Evaluating Nationwide Genetic Carrier Testing Begins Full Recruitment in Australia
Full-scale recruitment for an AUD$20 million (about $13.9 million U.S.) nationwide study to screen for severe genetic conditions has begun in Australia. Following the ... Read more -
Waiting for Fireflies
Dusk was settling in. The sun had clocked out for the day, and as it traveled farther and farther away toward its sleeping quarters, ... Read more -
Ella Skips a Level in Choir
Ella has always loved to sing. Because she can’t join physical activities or play an instrument, we decided to enroll her in the Young ... Read more -
Too Little Known About Care Needs of Adults with SMA, Study Says
A better understanding of the needs of adults with spinal muscular atrophy (SMA), best ensuring for their mental and social — as well as physical ... Read more -
Adapting to Cat Motherhood, SMA-style
“I know I’m on my period,” I told my dad, “because I’m crying at the mere possibility of Rey not knowing that I love ... Read more
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Researchers Discover Promising New Gene Therapy Approach for Spinal Muscular Atrophy
News
A new genetic approach to restore the expression of the gene that causes spinal muscular atrophy (SMA) in patients’ cells holds promise to effectively and permanently treat the disease, researchers suggest. The study, “Seamless genetic conversion of SMN2 to ... Read more