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...
The nasally administered benzodiazepine spray midazolam (Nayzilam; UCB, Smyrna, GA) has been approved by the Food and Drug Administration (FDA) for acute treatment of frequent seizure activity in people age 12 and up with epilepsy. The nasal spray is a single-use treatment that can be administered when a seizure cluster occurs. Midazolam is the first new medication approved for treating seizure clusters in more than 20 years in the US. In a randomized double-blind clinical trial (NCT01390220), a statistically significant higher proportion of seizure episodes treated with midazolam (n = 134)...
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...
Charisse Litchman, MD, FAHS; and Sirisha Sanamandra, MBBS
Gordon H. Baltuch, MD, PhD
Lauren Doyle Strauss, DO, FAHS; and Scott Otallah, MD