Diseases & Diagnoses
Choose any area of neurology to see curated news, articles, case reports, and more on that topic.
Patients & Caregivers
Find information and tools about neurological diseases to assist patients and caregivers.
The Food and Drug Administration (FDA) has approved onasemnogene abeparvovac-xioi (Zolgensma; Avexis, Bannockburn, IL) for the treatment of all types of spinal muscular atrophy (SMA) in children under age 2 years. Approved indications for onasemnogene, an SMN gene-replacement therapy, include all types of SMA. Treatment of presymptomatic children diagnosed through genetic screening is also approved. Not all states include testing for SMA in newborn screening panels, however. Onasemnogene is given intravenously in a single 1-time infusion. In 2 clinical trials (STRIVE [NCT03306277] and...
In a double-blind phase 2 study (NCT02975349), adult participants with multiple sclerosis (MS) who were treated with evobrutinib (EMD-Serono, Rockland, MA) (75 mg taken orally once or twice daily) had reduced numbers of T1 gadolinium enhancing (Gd+) lesions as early as 12 weeks after initiating treatment (Table 1). This reduction in T1 Gd+ lesions was maintained through week 48 of the study (Table 2). Participants treated with evobrutinib also had reduced annualized relapse rate (ARR) compared with those treated with placebo (Table 1), although this did not reach statistical...
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...
Andrea P. Lee, MD; Giulietta M. Riboldi, MD; Ilya Kister, MD; Jonathan E. Howard, MD; and Ritesh A. Ramdhani, MD
Aniket Natekar, MD, MSc; Malya Sahu, BSc; Hsiangkuo Yuan, MD, PhD; and Stephanie Nahas, MD, MSEd, FAHS, FAAAN
Regina Krel, MD; and Paul G. Mathew, MD, DNBPAS, FAAN, FAHS