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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...
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....
Post hoc analysis shows that participants who took 100 mg or 200 mg of lasmitidan had statistically significant increases in the rate of pain relief and freedom from the most bothersome symptom (MBS) (ie, nausea, photophobia, or phonophobia) (P < .001 starting as early as 30 minutes after administration. Participants who rated themselves as nonresponders, poor responders, or good responder to triptans did not have significant differences in their responses to 200 mg lasmiditan in any measure (freedom from pain at 2 hours, freedom from MBS, or pain relief). Participants who...
Ronald Devere, MD, FAAN
Umer Najib, MD, FAHS; Jessica Frey, MD; and David B. Watson, MD, FAHS, FAAN
Gordon H. Baltuch, MD, PhD