Harmony Begins New Phase 3 TEMPO Study of Wake-Promoting Therapy Pitolisant in Prader-Willi Syndrome
April 9th 2024The newly initiated phase 3 trial follows a successful phase 2 study where treatment with pitolisant resulted in greater improvement on the Epworth Sleepiness Scale for Children and Adolescents than placebo.
Marinus Reports Several Positive Trial Updates on Antiseizure Medication Ganaxolone
April 8th 2024Ganaxolone, a synthetic neurosteroid, has ongoing studies in tuberous sclerosis complex and super refractory status epilepticus, as well as upcoming research in Lennox-Gastaut syndrome and other epileptic encephalopathies.
Targeting Neurodegeneration to Impart Neuroprotection in Progressive MS: Shiv Saidha, MBBCh
April 4th 2024The professor of neurology at Johns Hopkins Medicine discussed the limited impact of current therapies on retinal atrophy rates in progressive multiple sclerosis at the 2024 ACTRIMS Forum. [WATCH TIME: 5 minutes]
Overcoming Access Barriers to Epilepsy Surgery: Hai Sun, MD, PhD
April 3rd 2024The associate professor of neurosurgery at Robert Wood Johnson Medical School talked about the superiority of surgical treatment for certain types of epilepsy and the barriers patients face to access this treatment approach. [WATCH TIME: 5 minutes]
Evaluating Givinostat's Efficacy and Safety in Duchenne Muscular Dystrophy: Marcello Paglione, PhD
April 3rd 2024The executive medical lead at Italfarmaco SpA talked about data from both animal models and clinical trials supporting the efficacity of givinostat in patients with Duchenne muscular dystrophy at MDA 2024. [WATCH TIME: 5 minutes]
NeuroVoices: Francesco Saccà, MD, PhD, on Crossing Over Dimethyl Fumarate in Friedriech Ataxia
April 3rd 2024The associate professor of neurology at the University of Naples discussed a recently enrolled study assessing the use of dimethyl fumarate, an approved therapy for relapsing MS and psoriasis, in patients with Friedreich ataxia.