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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…

The HBO series “The Last of Us” takes place in a dystopian world where zombie-like creatures roam the Earth 20 years after a mass infection wreaked havoc on humanity. Its main characters, Joel (Pedro Pascal) and Ellie (Bella Ramsey), travel across a post-apocalyptic America in an attempt…

A 23-day-old girl with spinal muscular atrophy (SMA) type 2, later treated with Spinraza (nusinersen), underwent successful surgical repair of heart defects, according to a new case report. The researchers noted that individuals with SMA may be perceived as risky surgical candidates, considering the poor prognosis for…

You know what they say. If something isn’t working, try something else. It’s perfectly logical when you think about it. Why sink time and energy into something that isn’t getting you results? But I’ve found that it’s hard to disinvest from something that, for all intents and purposes, should…

It’s hard to say exactly what impact rare disease awareness has had on our world. What has it taught us? Those of us with SMA and other rare diseases are keenly aware of the benefits that years of advocacy, research, fundraising, and education have brought to our disability…