Children with spinal muscular atrophy (SMA) who were diagnosed through newborn screening and treated early had better long-term independence and daily functioning, especially when supported by multidisciplinary care at home and at school, according to…
A preclinical study found that blocking the SMPD1 enzyme with available drugs such as the antidepressant clomipramine increases the levels of SMN, the protein missing in spinal muscular atrophy (SMA), potentially offering a new treatment strategy. “The strategy presented…
ARD-1676, a medication that works by tagging toxic proteins for destruction in cells, promotes clearance of the androgen receptor (AR) protein, which is faulty and clumps in spinal and bulbar muscular atrophy (SBMA), a study found. Earlier approaches…
For adults with spinal muscular atrophy (SMA), a genetic disease that progressively weakens breathing muscles, the approved treatment Spinraza (nusinersen) offers a crucial long-term benefit — stable lung function over several years. Data from a multi-center…
The U.S. Food and Drug Administration (FDA) has declined to approve a higher dose of nusinersen — the medication in Biogen’s long-approved therapy Spinraza — for the treatment of spinal muscular atrophy (SMA). According to Biogen, no…
An experimental oral combination of two therapies eased motor symptoms and prolonged survival in a mouse model of spinal muscular atrophy (SMA), a study shows. The study, “The effect of coadministration of D156844 and AR42 (REC-2282) on…
The use of salbutamol may safely improve upper limb strength, as well as provide other benefits, in people with spinal muscular atrophy (SMA), particularly among young children, according to data from a new meta-analysis — though…
A quantitative MRI scan, which shows detailed images of muscles, can be used to track changes seen with Spinraza (nusinersen) treatment in children with spinal muscular atrophy (SMA), according to a new study from China.
Offering children with spinal muscular atrophy (SMA) type 1 the opportunity to move around on their own using motorized, ride-on cars may boost their confidence, help them connect with others, and make them feel more included, a…
After Spinraza (nusinersen) treatment for slightly longer than one year, school-aged children with spinal muscular atrophy (SMA) showed improved motor function and more independence in tasks involving their arms and hands, a study from China has found.
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