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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 -
Raising a Puppy Is Hard but Rewarding Work
We recently welcomed a new addition to our family. Our adorable puppy, Thor, is a mix of Australian shepherd (75%) and blue heeler (25%). ... Read more -
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays ... Read more -
Plant Compound, Berberine, Seen to Clear Protein Clumps That Mark SBMA
Berberine, a plant-derived compound often used in Chinese medicine, is able to clear the toxic protein clumps found in neurodegenerative diseases such as spinal and ... 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