When you have a rare disease like SMA, there comes a point when you have to consciously choose to trust your doctors. Luckily for me, I grew up with a robust medical team. I had access to a variety of well-renowned medical systems, including my beloved Gillette Children’s, M…
Discussion
More than a year after the U.S. Food and Drug Administration (FDA) rejected the muscle-strengthening agent apitegromab for spinal muscular atrophy (SMA) due to manufacturing issues, the agency has approved the add-on treatment for use by certain people with the rare genetic condition. The therapy’s developer…
Discussion
My late friend and colleague Michael Morale liked to utter a phrase to me whenever I had an aquatic therapy session: “Get strong.” Michael spoke with a thick Texan accent and the utmost sincerity as he emphasized those words. He fully believed that…
Discussion
A European survey of people living with spinal muscular atrophy (SMA) has found that while treatment options have expanded since the first medicine was approved in 2017, patient journeys remain far from straightforward. Data from the 2025 European Patient Experience Survey on SMA Medicines, Access, and Treatment Journeys…
Discussion
A new blood test may offer a faster and simpler way to reliably diagnose most cases of spinal muscular atrophy (SMA), with results available in about one hour, a study showed. The LNA-PCR assay looks for homozygous SMN1 deletions, the most common SMA-causing mutation, in which a key section of…
Discussion
To say I didn’t attempt to set my complaint about a Sephora event up for success … would be a lie. If I was posting a video on TikTok, I obviously wanted people to see it. Time and effort are required to write a script, film, and edit,…
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