Immune Response to Zolgensma Can Be Common in Older SMA Children
Abnormal increases in liver enzymes levels — suggestive of liver damage — and drops in platelet counts are common among spinal muscular atrophy (SMA) patients over 8 months old following treatment with Zolgensma, according to a small real-world study in Germany. These...
Apitegromab Over 1 Year Bolsters Motor Abilities of Types 2 and 3: TOPAZ Trial
Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), safely and effectively improved or stabilized motor function in children and young adults with types 2 and 3 disease over one year, top-line data from the Phase 2 TOPAZ trial show....
Evrysdi Approved in Europe as First Oral, At-home Treatment
The European Commission has approved Evrysdi (risdiplam) as the first oral and at-home treatment for adults, children, and infants 2 months and older with nearly all types of spinal muscular atrophy (SMA). Eligible patients include those with a clinical diagnosis of...
Survey: Benefits of SMA Newborn Screening Outweigh Disadvantages
The benefits of newborn screening (NBS) for spinal muscular atrophy (SMA) outweigh its disadvantages and challenges, according to a survey of parents and healthcare providers of screen-positive newborns in Australia. Notably, both groups supported the implementation...HCP — SMA News Today
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