spinal muscular atrophy

  1. Reimbursement information on Spinraza (nusinersen)

    Over 50 countries have approved Spinraza (nusinersen), but not all have made it available to the patients who need it.

    stethoscope on money and med chart

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  2. A new gene therapy on the horizon for SMA

    Zolgensma (onasemnogene abeparvovec-xxxx) to receive priority review by FDA as a one-time treatment of type 1 spinal muscular atrophy (SMA).

    baby outside reaching toward viewer

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