According to my late mother, I was a doting 2-year-old caregiver during her pregnancy, determined to keep her from getting hurt. I adored my parents but was unimpressed with my newborn brother, Paul. Knowing I wasn’t keen on sharing the attention, my folks deemed my offer to push baby Paul…
Discussion
Last week, I had the opportunity to meet a woman who has influenced my life for as long as I can remember. I was starstruck and a little overwhelmed at the thought of talking to her. But when we met, she acted as though she was the one privileged to…
Discussion
Combining two approaches to address problems in astrocytes, cells that support nerve cells (neurons), may improve communication between motor neurons in spinal muscular atrophy (SMA), a new study suggests. Researchers increased levels of SMN, the protein deficient in SMA, in lab-grown astrocytes derived from patients. They also used forskolin,…
Discussion
Novartis has confirmed that there have been at least two reports of tumors that appear to have been caused by treatment with Zolgensma (onasemnogene abeparvovec-xioi), a gene therapy that is widely approved for infants and young children with spinal muscular atrophy (SMA). Details about these cases…
Discussion
Recently, I was teasing my brother about how he apparently doesn’t care about me anymore. (Don’t worry. This couldn’t be further from the truth.) The next day, I was scheduled for an iron infusion and was dreading it. Because I live with spinal muscular atrophy (SMA), maintaining my iron…
Discussion
Long-term treatment with Spinraza (nusinersen) was associated with early improvements in motor function followed by long-term stability in people with spinal muscular atrophy (SMA) types 2 and 3, according to a real-world study in Turkey. After an average follow-up of more than four years, 75% of patients with…
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