Scholar Rock is working to bring spinal muscular atrophy (SMA) treatment Isembyld (apitegromab-mstn) to patients worldwide, following U.S. Food and Drug Administration (FDA) approval of the infusion therapy as the first muscle-strengthening agent to treat the disease. “Our work does not stop with the US approval,” Akshay…
Discussion
After its success in the U.S., Scholar Rock is once again seeking approval in the European Union for its muscle-strengthening drug apitegromab —now launching as Isembyld in America — for spinal muscular atrophy (SMA). The developer has resubmitted an application to the European Medicines Agency (EMA)…
Discussion
Our prayers have recently been answered with the arrival of a new weekend night nurse, and it feels like the wheels of progress are finally turning on my journey with spinal muscular atrophy (SMA), hopefully in a big way. The nurse couldn’t have come at a better — or…
Discussion
Babies born in maternity wards across the West Midlands, in England, are now being tested for spinal muscular atrophy (SMA) as part of a new National Health Service (NHS) initiative launched on Oct. 1. The regional rollout marks the first phase of a broader effort to expand newborn screening…
Discussion
One of my favorite tees to wear is navy blue and says, “INSTALLING MUSCLES. PLEASE WAIT …” Underneath the text is a bright red loading bar about halfway done. It was a gift from my mother, a token of her hope that I’d one day grow stronger instead of weaker.
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John Muir once wrote, “The mountains are calling, and I must go.” Every once in a while, that quote resonates in my heart. I am a Carolina girl who grew up in the foothills of the Great Smoky Mountains, along the rim of the Blue Ridge Parkway, and I somehow…
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