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March 1, 2021

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  • Long-Term Treatment of Hereditary Amyloid Neuropathy with Patisiran

    Hereditary transthyretin-mediated amyloidosis with polyneuropathy is effectively treated by patisiran, a lipid nanoparticle ribonucleic acid interference that binds to transthyretin (TTR) messenger RNA and inhibits TTR production in the liver, resulting in reduction of the serum TTR concentration. Treatment effect has been sustained during a five-year open-label extension study.

  • Gluten/Celiac Disease Neuropathy

    Gluten neuropathy should be suspected in any patient who presents with a neuropathy and has a history of gastrointestinal disease. The diagnosis is made using blood serology plus intestinal mucosal biopsy.

  • Efficacy and Safety of Cannabidiol in Treatment of Focal Seizures Associated with Tuberous Sclerosis Complex

    Cannabidiol used as an add-on therapy for intractable focal seizures in patients with tuberous sclerosis complex was efficacious. Cannabidiol also had an acceptable adverse event profile, but frequently induced transient elevation of liver enzymes.

  • Spontaneous Intracranial Hypotension Syndrome

    A systematic review of spontaneous intracranial hypotension demonstrates heterogeneity in clinical and radiographic presentations. Diagnostic studies, such as brain imaging and lumbar puncture, may be unrevealing, and the clinician may have to rely on symptom patterns alone to make a diagnosis.

  • Stem Cell Transplantation for MS Treatment

    In this small study of the effect of mesenchymal stem cell treatment, both intravenous and intrathecal, in patients with progressive multiple sclerosis, the treatment was shown to be safe, and there was a trend showing some benefit in slowing disease progression, compared to the sham group. A larger randomized controlled trial is needed to confirm these early findings.