More than a year after the U.S. Food and Drug Administration (FDA) rejected the muscle-strengthening agent apitegromab for spinal muscular atrophy (SMA) due to manufacturing issues, the agency has approved the add-on treatment for use by…
Managing a career, dating, and air travel for individuals with spinal muscular atrophy (SMA) comes down to three key tools: open communication, smart energy management, and proactive planning. These strategies were discussed during a recent…
The successful implementation of a spinal muscular atrophy (SMA) carrier screening program in southern China shows the “feasibility and clinical utility” of such a program in the country, researchers said. The program tested thousands of people, allowing…
In spinal muscular atrophy (SMA), the death of nerve cells — which results in the progressive muscle weakness and wasting that characterize the rare disease — begins in wire-like nerve fibers known as axons,…
The U.S. Food and Drug Administration (FDA) has once again found problems at a manufacturing facility that was included on Scholar Rock‘s application seeking approval of apitegromab, its experimental muscle-strengthening agent for spinal…
In a few weeks, SMA News Today will host a free webinar exploring aspects of life with spinal muscular atrophy (SMA) that are rarely discussed in the doctor’s…
Registry studies can provide a valuable glimpse into how outcomes are evolving in rare diseases such as spinal muscular atrophy (SMA), but a lack of complete data can limit their usefulness, a study found. The researchers called for…
In infants diagnosed via newborn screening with spinal muscular atrophy (SMA), measures of nerve function may be a useful way to quickly evaluate disease activity and track how these young patients respond to SMA…
Different types of nerve cells respond differently when activity of a gene linked to spinal muscular atrophy (SMA) is reduced — and responses can vary even among nerve cells of the same type, according to a new…
August is SMA Awareness Month, and advocates around the world are gearing up to raise awareness about spinal muscular atrophy (SMA) by sharing stories, raising funds, and connecting as a community. SMA News Today will mark the month…
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