Showing 971-980 of 1043 results for "".
- Wakix Treatment for Excessive Daytime Sleepiness in Pediatric Patients with Narcolepsy Approvedhttps://practicalneurology.com/news/wakix-treatment-for-excessive-daytime-sleepiness-in-pediatric-patients-with-narcolepsy-approved/2470515/The Food and Drug Administration (FDA) has approved Harmony Biosciences’ supplemental New Drug Application (sNDA) for Wakix (pitolisant; Harmony Biosciences, Plymouth Meeting, PA) for the treatment of excessive daytime sleepiness (EDS) in pediatric patients (aged 6 to <18 years) with nar
- FDA Grants Orphan Drug Designation to AAV Gene Therapy Product for Malignant Gliomahttps://practicalneurology.com/news/fda-grants-orphan-drug-designation-to-aav-gene-therapy-product-for-malignant-glioma/2470368/NXL-004 (NeuExcell Therapeutics, Philadelphia, PA) has been granted Orphan Drug Designation (ODD) by the Food and Drug Administration (FDA). NXL-004 is the first investigational adeno-associated virus vector (AAV) gene therapy product developed as a potential treatment for malignant glioma to rec
- FDA Accepts New Drug Application for GA Depot to Treat Relapsing Multiple Sclerosishttps://practicalneurology.com/news/fda-accepts-new-drug-application-for-ga-depot-to-treat-relapsing-multiple-sclerosis/2470265/The Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for GA Depot 40 mg (Viatris, Canonsburg, PA; Mapi Pharma, Ness Ziona, Israel) to treat relapsing multiple sclerosis (RMS). This long-acting
- FDA Authorizes Phase 2/3 Trial Procession for Buntanetap in Alzheimer Diseasehttps://practicalneurology.com/news/fda-authorizes-phase-23-trial-procession-for-buntanetap-in-alzheimer-disease/2470042/The Food and Drug Administration (FDA) has authorized the phase 2/3 clinical study of buntanetap (ANVS401; Annovis, Berwyn, PA) in individuals with moderate Alzheimer disease (AD). Approval to further pursue the development of buntanetap in AD was requested after the submission of the p
- First Participant Dosed with Buntanetap in Phase 3 Trial for Early Parkinson Diseasehttps://practicalneurology.com/news/first-participant-dosed-with-buntanetap-in-phase-3-trial-for-early-parkinson-disease/2470008/In a phase 3 clinical trial for early Parkinson disease (PD) treatment, the first participant was dosed with buntanetap (ANVS401; Annovis, Berwyn, PA). Planned enrollment is 450 participants with early PD who will be randomly assigned to receive 10 or 20 mg buntanetap or placebo, on top of t
- Buntanetap Safe With Signals of Efficacy for Alzheimer Disease and Phase 3 Trials Are Being Initiatedhttps://practicalneurology.com/news/buntanetap-safe-with-signals-of-efficacy-for-alzheimer-disease-and-phase-3-trials-are-being-initiated/2469994/In a phase 2 trial (NCT04524351) in people with early Alzheimer disease (AD) or Parkinson disease (PD) treatment with 5 to 80 mg/day oral buntanetap (ANVS401; Annovis, Berwyn, PA) for 25±2 days had no adverse events. These resul
- Analyses Show High Effect Sizes and Low Numbers Needed to Treat With Pitolisant for Narcolepsyhttps://practicalneurology.com/news/analyses-show-high-effect-sizes-and-lows-numbers-needed-to-treat-with-pitolisant-for-narcolepy/2469925/In a post hoc analysis, pitolisant (Wakix; Harmony Biosciences, Plymouth Meeting, PA) reduced excessive daytime sleepiness (EDS) and cataplexy in adults with narcolepsy and severe symptom burden. The effects of pitolisant were assessed with the Cohen’s d test, a measure of effect size, and
- Translingual Neuromodulator to Assist Ambulation in Multiple Sclerosis Now Available in UShttps://practicalneurology.com/news/translingual-neuromodulator-to-assist-ambulation-in-multiple-sclerosis-now-available-in-us/2469898/Helius Medical Technologies (Newtown, PA) has announced that their translingual neuromodulation (portable neuromodulation stimulator [PONS]) device, shown to assist walking in people with multiple sclerosis (MS), is now available in the US. The PONS device is a portable, nonimplant neuromod
- Long-Term Efficacy and Safety of Ganaxolone for Treatment of Cyclin-Dependent Kinase-Like 5 Deficiency Disorder—A Developmental Encephalopathic Epilepsyhttps://practicalneurology.com/news/long-term-efficacy-and-safety-of-ganaxolone-for-treatment-of-cyclin-dependent-kinase-like-5-deficiency-disorder-a-developmental-encephalopathic-epilepsy/2469875/Ganaxolone (Ztalmy; Marinus Pharmaceuticals, Radnor, PA) was recently approved for adjunctive treatment of cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in people, age 2 years or more. CDD, which can be diagnosed with a genetic test, is among the developmental
- FDA Approves Ganaxolone for Treatment CDKL5 Deficiency Disorder, a Rare Genetic Epilepsyhttps://practicalneurology.com/news/fda-approves-ganaxolone-for-treatment-cdkl5-deficiency-disorder-a-rare-genetic-epilepsy/2469857/The Food and Drug Administration (FDA) has approved ganaxolone (Ztalmy; Marinus Pharmaceuticals, Radnor, PA) for treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency, a rare genetic epilepsy. Ganaxolone is an oral suspension and was approved for individuals, age