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3 weeks ago | 
smanewstoday.com | Marisa Wexler |Alyssa Silva |Andrea Lobo
A tablet formulation of Evrysdi (risdiplam) for spinal muscular atrophy (SMA) recently approved in the U.S. is designed to give patients a new option, a Genentech official said.
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1 month ago | 
smanewstoday.com | Marisa Wexler |Alyssa Silva |Helen Baldwin
The muscle-strengthening therapy apitegromab is being considered for approval by the U.S. Food and Drug Administration (FDA) as an add-on treatment for spinal muscular atrophy (SMA), with a decision expected around the start of fall. Scholar Rock, the company developing apitegromab, applied seeking FDA approval earlier this year. This week, the FDA granted priority review to the application and set Sept. 22 as its decision date, the company announced.
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1 month ago | 
smanewstoday.com | Marisa Wexler |Margarida Maia |Katherine Poinsatte |Alyssa Silva
A higher dose of Spinraza (nusinersen) may be more effective than the currently approved dosing schedule in people with spinal muscular atrophy (SMA) for maintaining motor function, according to data from the DEVOTE clinical trial.
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1 month ago | 
smanewstoday.com | Alyssa Silva |Steve Bryson |Sherry Toh |Brianna Albers
Every now and then, memories from my childhood hit me in the most unexpected ways, which is surprising because I don’t remember much from years past. Alas, my parents were cleaning out our basement recently when they came across a large trash bag filled with some of my stuffed animals. Right on top was a stuffed horse I hadn’t seen in decades. The memories it still held sucker-punched me right in the gut and transported me back in time to a moment when life felt so heavy.
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1 month ago | 
smanewstoday.com | Marisa Wexler |Alyssa Silva
Apitegromab, a muscle-strengthening therapy up for approval in the U.S., led to gains in motor function in a clinical trial for people with spinal muscular atrophy (SMA) who took disease-modifying therapies. Data from the trial, called SAPPHIRE (NCT05156320), formed the basis of Scholar Rock‘s recent application asking the U.S. Food and Drug Administration to approve apitegromab for SMA.
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1 month ago | 
smanewstoday.com | Brianna Albers |Lindsey Shapiro |Alyssa Silva |Sherry Toh
Even with SMA, my checkups have, for the most part, become pretty routine. Every six months, I drive 30 minutes to my nearest specialty clinic. While there, I see various doctors, each with their own unique vibe. My neurologist catches me up on all the books he’s read recently. My complex care doctor puts me at ease with his sunny smile and easygoing demeanor. My wheelchair guy (for lack of a better term) jokes good-naturedly with my dad about manly things like nuts, bolts, and Allen wrenches.
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1 month ago | 
smanewstoday.com | Marisa Wexler |Steve Bryson |Ari Anderson |Alyssa Silva
The Muscular Dystrophy Association‘s (MDA) annual Clinical & Scientific Conference kicked off over the weekend, bringing together patients, caregivers, researchers, clinicians, academics, advocates, and industry leaders to discuss the latest in science and care related to a range of neuromuscular diseases. This year’s conference will bring together hundreds of participants from 39 countries, Morgan Roth, chief marketing officer of the MDA, told Bionews, the parent company of this site.
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1 month ago | 
smanewstoday.com | Kevin Schaefer |Margarida Maia |Alyssa Silva
When the movie version of “Wicked” hit theaters last fall and skyrocketed to critical and commercial success, musical theater enthusiasts everywhere felt validation. The blockbuster adaptation of the hit musical struck a chord with devoted fans, casual viewers, and anyone with a passing familiarity with “The Wizard of Oz” mythos. A while back, I wrote about my history with the stage musical, noting what I love about it as well as some of the show’s problematic depictions of disability.
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1 month ago | 
smanewstoday.com | Marisa Wexler |Lindsey Shapiro |Alyssa Silva |Steve Bryson
SMA Europe is funding five new research projects that are intended to advance understanding of spinal muscular atrophy (SMA) and guide the development of new therapies. The funding comes as part of SMA Europe’s 12th Call for Research proposals, which the advocacy organization runs every other year to support SMA research.
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1 month ago | 
smanewstoday.com | Jasmine Ramos |Kevin Schaefer |Halsey Blocher |Alyssa Silva
A few weeks ago, my mother injured her foot, temporarily limiting her mobility. As she struggled to get around, relying on others for assistance, she looked at me and said, “I never realized how hard this is.” That moment stuck with me — not because I was surprised, but because she got a small glimpse into my world for the first time. Living with spinal muscular atrophy (SMA), I’ve faced mobility challenges my entire life. While my mother’s injury will heal, my condition is permanent.
