The U.S. Food and Drug Administration (FDA) has shared new details concerning reports of cancer developing following treatment with gene therapy for spinal muscular atrophy (SMA). Last week, Novartis confirmed that there have been nine cases of tumors developing after treatment with its gene therapy…

When someone asks me what a good day looks like, my answer probably isn’t what they expect. It doesn’t necessarily involve accomplishing a long list of tasks, doing everything independently, or having a day when my disability doesn’t get in the way. For me, a good day is much simpler.

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…

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…

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,…