Showing 2011-2020 of 2123 results for "".
- First Participant Enrolled in Phase 3 Trial of Gaboxadol for Angelman Syndromehttps://practicalneurology.com/news/first-participant-enrolled-in-phase-3-trial-of-gaboxadol-for-angelman-syndrome/2469019/The first participant has been enrolled and randomly assigned to a trial arm in the phase 3 NEPTUNE trial (NCT02996305) of gaboxadol (OV101; Ovid Therapeutics, New York, NY) for Angelman syndrome (AS). Approximately 60 childr
- Extended-Interval Dosing of Natalizumab Efficacy in Retrospective Propensity Score Analysis of Real-World Datahttps://practicalneurology.com/news/extended-interval-dosing-of-natalizumab-efficacy-in-retrospective-propensity-score-analysis-of-real-world-data/2469016/Using propensity score analysis in which individuals who had extended-interval dosing (EID) after at least 1 year of standard-interval dosing (SID) of natalizumab (Tysabri; Biogen, Cambridge, MA) were matched with individuals treated with standard-interval dosing (SID) for comparison of outcomes.
- Natalizumab Treatment Reduced Disability With No Cases of PML in Anti-JC-Virus Antibody Negative Multiple Sclerosishttps://practicalneurology.com/news/natalizumab-treatment-reduced-disability-with-no-cases-of-pml-in-anti-jc-virus-antibody-negative-multiple-sclerosis/2469010/In the STRIVE clinical trial (NCT01485003),4 years of treatment with natalizumab (Tysabri; Biogen, Cambridge, MA) reduced disability and annualized relapse rates (Table).
- First Participant Enrolled in Observational Study of Children and Teens With Dravet Syndromehttps://practicalneurology.com/news/first-participant-enrolled-in-observational-study-of-children-and-teens-with-dravet-syndrome-1/2468998/The first person has been enrolled in the BUTTERFLY observational study sponsored by Stoke Therapeutics (Bedford, MA). The BUTTERFLY study is a 2-year observationa
- Faster—and Free—Genetic Testing for Spinal Muscular Atrophyhttps://practicalneurology.com/news/fasterand-freegenetic-testing-for-spinal-muscular-atrophy/2468996/A new genetic test for spinal muscular atrophy (SMA) provides results in 4 days vs the 21 day wait previously needed. Early diagnosis is essential for early treatment, shown to slow disease progression, prolong survival by years, and improve or stabilize loss of motor function. Of note, the test
- Phase 1 Studies Begin for EPX-100 for Dravet’s Syndromehttps://practicalneurology.com/news/phase-1-studies-begin-for-epx-100-for-dravets-syndrome/2468983/The Food and Drug Administration has approved the Investigational New Drug Application for EPX-100 (Epygenix Therapeutics, Inc., Paramus, NJ), allowing a phase 1 study to being. This phase 1 placebo-controlled, 2-period study of EPX-100 will test safety and pharmacokinetics of escalating single a
- Phase 3 Trial of Fenfluramine for Lennox-Gastaut Syndrome Completes Enrollmenthttps://practicalneurology.com/news/phase-3-trial-of-fenfluramine-for-lennox-gastaut-syndrome-completes-enrollment/2468948/Enrollment of 263 individuals with Lennox-Gastaut syndrome (LGS) has been completed for a phase 3 clinical (NCT03355209) trial of fenfluramine (Fintepla; Zogenix, Emeryville, CA). The primary outcome measure is decreased seizure freque
- Potential Redefinition of “Unresponsive” for People in Hospital With Brain Injurieshttps://practicalneurology.com/news/potential-redefinition-of-unresponsive-for-people-in-hospital-with-brain-injuries/2468943/The inability to follow a simple command, such as “move your hand” has been termed unresponsive and thought to prognosticate poor neurologic outcomes for those with brain injury. It has not been possible to elucidate whether this status arises from sensory, motor, or cognitive deficit
- OnabotulinumtoxinA Approved for Treatment of Children with Upper Limb Spasticityhttps://practicalneurology.com/news/onabotulinumtoxina-approved-for-treatment-of-children-with-upper-limb-spasticity/2468936/The Food and Drug Administration (FDA) has approved a new indication for onabotulinumtoxinA (Botox; Allergan, Madison, NJ)—treatment of upper limb spasticity in children, age 2 to 17 years. The decision came after a 6-month priority review, which istypically granted to therapies with potent
- Phase 2 Study of Potential Acute Ischemic Stroke Treatment Planned for 2019https://practicalneurology.com/news/phase-2-study-of-potential-acute-ischemic-stroke-treatment-planned-for-2019/2468930/A phase 2 proof-of-concept study will begin in the US later this year for LT3001 (Lumosa Therapeutics, Taipei, Taiwan), a novel therapy for treating acute ischemic stroke (AIS). Enrollment of participants for the phase 2 study in Taiwan will begin in 2020. Full enrollment is expected to take 2 ye