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Researchers at Cedars-Sinai are working to develop a potential stem cell treatment for three teenage siblings in California with an extremely rare form of spinal muscular atrophy (SMA) for which there are…

Our third baby, Jeffrey, arrived May 18, 1997, three days after my 43rd birthday. He joined his siblings, Matthew, then 10, and Katie, then 7. Although the pregnancy was unplanned, my husband, Randy, and I…

A caregiver recently told me that the more she spends time helping me, the more she learns what I care most about. This longtime friend already knows that I care about faith, relationships, stories, advocacy, and…

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…

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…

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…

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…

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…

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…

Because I live with spinal muscular atrophy (SMA), I have used a power wheelchair for most of my life. As far as I can recall, I have had seven different chairs, each one unique…