Showing 951-960 of 1043 results for "".
- Losmapimod Improves Function and Slows Progression of Facioscapulohumeral Muscular Dystropyhttps://practicalneurology.com/news/losmapimod-improves-function-and-slows-progression-of-facioscapulohumeral-muscular-dystropy/2469622/Although the phase 2b ReDUX4 study (NCT04264442) of losmapimod (Fulcrum Therapeutics, Cambridge, MA) for treatment of facioscapulohumeral muscular dystrophy (FSHD) did not meet prespecified outcomes, clinical improvements were seen. Pa
- Mixed Sodium Oxybates Improve Idiopathic Hypersomniahttps://practicalneurology.com/news/mixed-sodium-oxybates-improve-idiopathic-hypersomnia/2469574/In a phase 3 study (NCT03533114) of mixed salt oxybates (MSO) (Xywav; Jazz Pharmaceuticals, Philadelphia, PA) for treatment of idiopathic hypersomnia (IH) showed improvement on multiple measures. All participants first received tr
- FDA Approves Expanded use of Pitolisant for Treatment of Cataplexy in Adults With Narcolepsyhttps://practicalneurology.com/news/fda-approves-expanded-use-of-pitolisant-for-treatment-of-cataplexy-in-adults-with-narcolepsy/2469405/The Food and Drug Administration (FDA) approved pitolisant (Wakix; Harmony Biosciences Plymouth Meeting, PA)—treatment of cataplexy in adults with narcolepsy. Pitolisant is the first and only treatment not categorized as a controlled substance that is approved by the FDA for treating sympto
- New Mixed-Salt Oxybates Effective for Treatment of Hypersomnia in Adultshttps://practicalneurology.com/news/new-mixed-salt-oxybates-effective-for-treatment-of-hypersomnia-in-adults/2469398/A phase 3 study (NCT03533114) has evaluated use of mixed calcium-, magnesium-, potassium-, and sodium-oxybates (Xywav; Jazz Pharmaceuticals, Philadelphia, PA) for treatment of idiopathic hyp
- Phase 3 Trial of Ganaxalone for Refractory Status Epilepticus Being Initiatedhttps://practicalneurology.com/news/phase-3-trial-of-ganaxalone-for-refractory-status-epilepticus-being-initiated/2469387/Now that protocol-specific questions from the FDA have been answered, a phase 3 study (NCT03350035) of ganaxolone (Marinus, Radnor, PA) for refractory status epilepticus (RSE) will be initiated. Enrollment in the RAISE trial is schedul
- Translingual Neuromodulation Improves Chronic Balance Deficit in Clinical Trialhttps://practicalneurology.com/news/translingual-neuromodulation-improves-chronic-balance-deficit-in-clinical-trial/2469249/The TBI-001 trial (NCT01847755) found that translingual neurostimulation with a portable device (PoNS; Helius Medical Technologies, Newtown, PA) provided significant balance improvement in f
- Study of Ganaxalone for Children With CDKL5 Deficiency Disorder Currently Enrolled 100 Participantshttps://practicalneurology.com/news/study-of-ganaxalone-for-children-with-cdkl5-deficiency-disorder-currently-enrolled-100-participants/2469169/The Marigold study (NCT03572933) has obtained a 100 participant enrollment as of today. The Marigold study is a pivotal phase 3 study evaluating ganaxolone (Marinus Pharmaceuticals, Radnor, PA) in
- Phase 2 Glioblastoma Multiforme Study Exhibits Progression Free Survivalhttps://practicalneurology.com/news/inovio-exhibits-progression-free-survival-in-phase-2-glioblastoma-multiforme-study/2469068/Positive interm results of a T cell-activating immunotherapy (INO-5401; Inovio Pharmaceuticals, Plymouth Meeting, PA) were seen in a phase 2 study (NCT03491683) of a T cell-activating immunotherapy for treatment of newly diagnosed gliob
- Novel Oxybate With 92% Less Sodium Effectively Treated Cataplexy and Excessive Daytime Sleepiness of Narcolepsy in Phase 3 Trialhttps://practicalneurology.com/news/novel-oxybate-with-92-less-sodium-effectively-treated-cataplexy-and-excessive-daytime-sleepiness-of-narcolepsy-in-phase-3-trial/2469035/In a phase 3 double-blind placebo-controlled randomized-withdrawal study (NCT03030599) a novel oxybate (JZP-258; Jazz Pharmaceuticals, Philadelphia, PA) was effective for treatment of cataplexy and excessive daytime sleepiness (EDS) in
- FDA Approves Pitolisant-First-in-Class Nonscheduled Treatment for Excessive Daytime Sleepiness in Adults With Narcolepsyhttps://practicalneurology.com/news/fda-approves-pitolisant-first-in-class-nonscheduled-treatment-for-excessive-daytime-sleepiness-in-adults-with-narcolepsy/2468991/Approval of pitolisant (Wakix; Harmony Biosciences, Plymouth Meeting, PA) by the Food and Drug Administration (FDA) makes it the first and only approved medication for treatment of excessive daytime sleepiness (EDS) in adults with narcolepsy that is not scheduled as a controlled substance by the