Diseases & Diagnoses
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The Food and Drug Administration (FDA) has approved amifampridine (Ruzurgi; Jacobus Pharmaceutical Company, Princeton, NJ) for treating Lambert-Eaton myasthenic syndrome (LEMS) in patients age 6 to 17 years. This is the first FDA approval of a therapy specifically for pediatric patients with LEMS, a rare autoimmune neuromuscular junction disorder. Use of amifampridine in pediatric patients is supported by clinical studies in adults with LEMS, pharmacokinetic data in adult patients, pharmacokinetic modeling and simulation to identify the dosing regimen in pediatric...
The Alzheimer’s Clinical Trials Consortium (ACTC) has announced 2 studies of combination therapy of elenbecestat (Eisai, Woodcliff Lake, NJ) and BAN2401(Eisai, Woodcliff Lake, NJ and BioArctic, Stockholm, Sweden) for potential prevention of Alzheimer’s disease (AD). The ACTC was formed with funding from the National Instute on Aging (NIA). In the A3 study, participants without cognitive impairment or elevated amyloid levels who are at risk for amyloid-baccumulation will be randomly assigned to receive elenbecestat alone (2 doses being tested) or placebo. Elenbecestat is...
Over 7,500 individuals have been or are being treated with nusinersen (Spinraza; Biogen, Cambridge, MA). Across clinical trials and the real-world setting, there are now 7 years of data and experience showing that nusinersen has clinical efficacy and safety. In the NURTURE study (NCT02386553) in which presymptomatic infants likely to develop spinal muscular atrophy type one (SMA1) or type 2 (SMA2) were treated with nusinersen, children continue to develop and gain typical motor milestones. After 26 months of treatment, all infants could sit without support; 88% could walk with support and 77%...
Emmanuelle A. D. Schindler, MD, PhD; and Christopher Gottschalk, MD
Regina Krel, MD; and Paul G. Mathew, MD, DNBPAS, FAAN, FAHS
Gordon H. Baltuch, MD, PhD