2 Topic Commentaries

FDA approves muscle-targeted SMA therapy

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  Thomas O. Crawford, MD

  Pediatric neurology; neuromuscular disorders

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  Johns Hopkins Medicine (Johns Hopkins University School of Medicine)

  Based on our findings, apitegromab has the potential to positively impact people with SMA, particularly those with weakened muscles who are unable to walk, allowing them to build muscle again and improve quality of life.

  Source
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  Elizabeth Kichula, MD, PhD

  Pediatric neurology; neuromuscular medicine

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  Children’s Hospital of Philadelphia

  While SMA remains a serious and life-threatening disorder, Spinraza and Zolgensma are changing the outlook for patients. They can stop progression of disease, making early diagnosis and rapid treatment critical to ensuring the best outcomes. While they are still not cures, they allow for continued motor improvement, particularly in the setting of supportive families and a commitment to rehabilitative therapy.

  Source