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[Editor’s note: An earlier version of this story did not specify in its opening statement that Spinraza’s use in this proposed trial would follow Evrysdi’s use.] Biogen plans to launch a Phase 3b trial into the safety and efficacy of a higher dose of Spinraza (nusinersen) in children,…
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Have you ever looked back on the person you used to be and thought, “That was a lifetime ago!” When I think this way, I look at it from two different perspectives. On one hand, yes, my past self seems light-years away. On the other hand, that other me seems…
Being surrounded by tranquility and stunning scenery in the North Carolina mountains is a true blessing. A bonus is that our two grandchildren live down the road! Clara, 5, and James, 3, entertain us all with their endless antics. It’s great fun for my husband, Randy, and me to spy…
Blood levels of neurofilament light chain (NfL) — a marker of nerve cell damage — in children and adolescents with spinal muscular atrophy (SMA) are significantly higher than those of age-matched healthy peers and associate with disease severity, a study found. NfL blood levels also reliably reflect levels in…
A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…
When it comes to vendettas, I have two. One is against Sigmund Freud. Freud, the “father” of psychology, who’s known for his unbelievably misogynistic views. Google “Freud misogyny” and you’ll get a laundry list of jeremiads decrying his influence on the mental health profession. Some of my favorites include…
A naturally occurring form of the androgen receptor protein — which scientists have named AR45 because of its size — may be useful for treating spinal and bulbar muscular atrophy (SBMA), a rare form of adult-onset spinal muscular atrophy, a new study suggests.
About a decade ago, I woke to the sight of dozens of butterflies bathed in gentle light above my head. I wasn’t expecting that. When I lost consciousness, my mom and I had just arrived at the hospital in an ambulance after being rescued from our home by…
Changes and hardships can often lead to growth and beauty, writes columnist Halsey Blocher, similar to a butterfly’s metamorphosis.
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Becoming educated and informed about spinal muscular atrophy is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your SMA journey.
There is no cure for SMA yet, but there are treatments that can help manage the disease. Learn more about current therapeutic approaches here.
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