Columns

“A hero — not the hero we deserved, but the hero we needed.” This line from a Batman movie aptly describes the life and legacy of Judy Heumann. The prominent disability rights activist, who died March 4 at the age of 75, remains a hero in…

If there’s one thing my dad and I can agree on, it’s that mornings are the worst. My mom, on the other hand, is a veritable morning person. She wakes up energized and ready to conquer the world. By the time I drag my sorry butt out of…

I still remember exactly what I was doing when news broke that Evrysdi (risdiplam) had been approved by the U.S. Food and Drug Administration (FDA) in August 2020. Up until that point, it had been a pretty ordinary day. It took only a moment to become…

A year of treatment with Evrysdi (risdiplam) was safe and boosted levels of SMN — the protein that’s deficient in spinal muscular atrophy (SMA) — among previously treated patients, according to published Phase 2 trial data. The safety profile was deemed similar to that observed in trials testing…

Children with spinal muscular atrophy (SMA) type 1 who start treatment with approved therapies after their symptoms emerge may be more likely to show below average cognitive, communication, and motor skills than those who start treatment before their symptoms, a study found. Cognitive function appeared to be most…

Avenue Therapeutics has won the exclusive rights to develop and commercialize AJ201, AnnJi Pharmaceutical’s experimental treatment for spinal and bulbar muscular atrophy (SBMA), in certain regions of the globe under the terms of a new licensing agreement. Specifically, the agreement lists the U.S., Canada, the European…

TREAT-NMD and Aetion have formed a partnership to deliver real-world evidence and promote the development of new treatments for people with rare neuromuscular diseases, including spinal muscular atrophy (SMA). The projects will combine the expertise of the TREAT-NMD global registry network, which collects de-identified data from…

Among the many SMA symptoms I live with every day, fatigue has permanently staked out its territory at the top of the list. I am perpetually tired. There are days when my fatigue gets the best of me, but more often than not, I’ve learned to live with…

Hello, March! Here in the North Carolina mountains, daffodils optimistically poke through the ground; impatient ones bloomed last week. Trees are budding, and flower beds are sporting a bit of green. Forager bees are gathering pollen for the baby bees. With all this popping up and out, you’d think…

More than 1 in 4 ambulatory spinal muscular atrophy (SMA) patients experienced clinically meaningful improvements in walking ability after being treated with Spinraza (nusinersen), according to an analysis of real-world findings in Europe. “Our data demonstrate a positive effect of [Spinraza] treatment on motor function in ambulant pediatric…