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Results from multiple trials of SMN1gene-replacement therapy (Zolgensma; Avexis, Bannockburn, IL) show promise for single-dose treatment of spinal muscular atrophy (SMA). In the STRIVE phase 3 study for treatment of spinal muscular atrophy, 22 infants under age 6 months, diagnosed genetically (biallelic SMN1 mutations/deletions, 2 SMN2 copies), were treated. Of the 15 babies treated more than 13.6 months or who discontinued the study before then, 13 (87%) survived without needing permanent ventilation, in contrast to the 25-50% survival rate for children with SMA when untreated....
The National Institute on Aging (NIA) has provided a 4-year grant for a phase 2 study of T3D-959 (T3D Therapeutics, Research Triangle Park, NC), a novel, metabolic-focused treatment for Alzheimer’s disease (AD). The PIONEER study, which will begin patient dosing in early 2020, is a double-blind placebo-controlled efficacy and safety study that will enroll up to 252 adults with mild-to-moderate AD. Participants in the study will receive 1 of 3 different oral doses of T3D-959 or placebo for 24 weeks. In a phase 2a study (NCT02560753), 34 participants with...
Treatment with inebilizumab (VielaBio, Gaithersburg, MD) reduced relapses of neuromyelitis optica spectrum disorder (NMOSD) by 77% after 28 weeks of treatment. Response to treatment with inebilizumab for NMOSD was safe and effective such that the trial was terminated early as it was deemed unethical not to treat participants in the placebo arm of a phase 3 trial (NCT02200770). After this judgement was made, all participants in the placebo arm began receiving inebilizumab on an open-label extension basis. Treatment with inebilizumab also reduced worsening from baseline score on...
Ronald Devere, MD, FAAN
Gordon H. Baltuch, MD, PhD
Charisse Litchman, MD, FAHS; and Sirisha Sanamandra, MBBS