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…
Discussion
It’s almost impossible for me to go places without silently analyzing the accessibility. I do it in my own city, but even more when we travel. After living with SMA for 29 years, it’s just natural to look around and quickly determine whether I can actually be a part…
Discussion
A few years ago, November was when writers worldwide would transform into hermits. We would hunker down and attempt to write 50,000 words by the end of the month — the equivalent of a novel. Rarely could we be seen during this time, except when fetching food and drink and…
Discussion
I often write about the caregivers who help me live a full and meaningful life with spinal muscular atrophy (SMA), and it is the primary theme of this column. Since I’ve lived most of my adult life away from home, I usually focus on the many wonderful friends in…
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