Spinal muscular atrophy (SMA) is an inherited condition that places a tremendous burden on patients, families, caregivers, and healthcare providers, with patients experiencing a wide range of complications and symptoms that are detrimental to day-to-day life. Although SMA management has historically consisted of supportive care, the therapeutic landscape has changed dramatically following the recent approval of transformative medications, particularly disease-modifying therapies (DMTs) that improve motor function and survival. In this educational activity, experts in SMA seek to bring clinicians up-to-date by discussing recent advances in screening for SMA, as well as need-to-know information on newer DMTs, such as relevant clinical efficacy and safety data and recommendations for treatment selection.
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