Radiologist Resource Pages

  • Administering Zolgensma

    Zolgensma, or onasemnogene abeparvovec, is an adeno-associated virus vector-based gene therapy that was approved by the FDA in May 2019. Given the newness of this therapy, it is important that healthcare providers are educated on specific information regarding the administration of Zolgensma so that they can optimize ...Read more

  • Patient and Parent Perspectives on the Use of Nusinersen for Spinal Muscular Atrophy

    At the end of 2016, the U.S. Food and Drug Administration (FDA) approved nusinersen for the treatment of spinal muscular atrophy (SMA).1 Because of the high threshold for FDA approval, an abundance of data on nusinersen and its physiological effects on SMA patients has been collected in recent years. However, given ...Read more

  • How to Administer Nusinersen in Adolescents¬†

    Nusinersen is the first treatment available for children with spinal muscular atrophy (SMA). Before its approval in 2016, the U.S. Food and Drug Administration had not approved the use of any drug for SMA treatment.1 While the introduction of this new drug marks significant progress in SMA therapy and provides new ...Read more

  • Spinal Muscular Atrophy: Awareness and Attitudes

    There is a paucity of information on awareness of and attitudes towards spinal muscular atrophy (SMA) and medical approaches to the disease.1,2 However, research into what is known about SMA has revealed that the public is largely unfamiliar with SMA, and even physicians and other healthcare providers have a limited ...Read more

  • Alternate Nusinersen Administration Strategies

    In the pivotal clinical trials that support FDA approval, nusinersen was administered via intrathecal injection. However, this approach requires that patients receive multiple intrathecal injections separated in time by weeks. During each infusion, the clinician must safely access the intrathecal space, which can be ...Read more

  • Administering Nusinersen

    Nusinersen (Spinraza) is the first FDA-approved antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in adults and children.1,2 The oligonucleotide is administered intrathecally2; thus, administration is not as straightforward as an intravenous infusion. As such, healthcare ...Read more

  • Spinal Muscular Atrophy: Electromyography

    Spinal Muscular Atrophy (SMA) is a progressive neuromuscular disease associated with typically proximal muscle weakness and atrophy due to degeneration of the anterior horn cells of the spinal cord.1  Prior to genetic testing as the gold standard diagnostic method for SMA, electromyography (EMG) was used widely ...Read more

  • Spinal Muscular Atrophy: Magnetic Resonance Imaging

    Spinal Muscular Atrophy (SMA) is a progressive neuromuscular accompanied by significant typically proximal muscle weakness and atrophy due to degeneration of the anterior horn cells of the spinal cord.1  Diagnosis of SMA does not require magnetic resonance imaging (MRI), but neuroimaging of the central nervous ...Read more

  • Chest Physiotherapy and Pulmonary Care in the Acutely Ill Child

    Children with spinal muscular atrophy (SMA) often have difficulty with breathing and maintaining blood oxygenation in periods of relative wellness. As such, they often have limited reserve pulmonary and cardiovascular function. Thus during an acute illness, especially a respiratory illness, chest physiotherapy and ...Read more

  • Chest Physiotherapy in the Well Child

    Respiratory problems are the leading cause of acute illness and chronic disability in infants and children with spinal muscular atrophy (SMA). Among children with SMA type I and type II, respiratory issues are the most common cause of death. On the other hand, when started early, proactive, noninvasive respiratory ...Read more