The other weekend, I was invited to a golf tournament. It was a gorgeous day, and I rarely turn down an invitation for a good time, so naturally, I channeled my inner Rory Gilmore, donned my cutest golfing outfit, and asked my official caddy to tote my clubs…
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The U.S. Food and Drug Administration (FDA) has declined to approve apitegromab as a treatment for spinal muscular atrophy (SMA). The FDA said it could not approve developer Scholar Rock‘s application at this time because of observations identified during a routine general site inspection of Catalent…
Various ethical and social factors influence care decisions for families affected by spinal muscular atrophy (SMA) in this new era of disease-modifying therapies (DMTs), according to a Canadian study. In interviews, caregivers reported that a range of factors, including financial resources and their expectations of treatment outcomes, influenced their…
Words always come to me when I need them. Lines by Ada Limón, Anne Boyer, and Marie Howe act as life preservers, guiding me back to my center. When life gets tough, I always find myself at the feet of Julian of Norwich. “All shall be well,” she says,…
Biogen has acquired Alcyone Therapeutics and will continue to develop ThecaFlex DRx, a device intended to ease patient experience and accessibility when delivering certain medicines into the spinal canal. The ThecaFlex DRx System is initially being evaluated with Spinraza (nusinersen) in people with spinal muscular atrophy (SMA),…
Everyone experiences times when they feel overwhelmed by problems to fix, regardless of whether they have SMA, as I do. My life right now is a work in progress, and there’s nothing wrong with that. I’m currently addressing two main issues: frequent illness and a lack of nursing coverage.
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