Investigational DMD Gene Therapy Reports Positive Preliminary Results in Phase 1b
July 1st 2019PF-06939926 is a recombinant adenoassociated virus serotype 9 (AAV9) capsid carrying mini-dystrophin, a shortened version of the human dystrophin gene, under the control of a muscle-specific promoter.
Zogenix to Resubmit Fenfluramine NDA for Dravet Syndrome
July 1st 2019After receiving a refusal to file letter and undergoing a Type A meeting with the FDA, Zogenix has announced that it plans to resubmit an NDA for fenfluramine (Fintepla) for the treatment of seizures associated with Dravet syndrome in Q3 of 2019.
Addressing Serious Neurologic Conditions in Men
June 28th 2019The clinical assistant professor of neurology and neurosurgery at the University of Texas spoke about the staggering number of men who forgo seeing physicians for serious conditions and how the clinical community can help address the challenge.
In Intractable Epilepsy, Rufinamide is Well-Tolerated and Effective
June 26th 2019Data suggest that rufinamide is capable of aiding in the control and reduction of seizures, as well as the possible achievement of seizure-freedom in pediatric patients with genetic/metabolic, hypoxic-ischemic, structural, and other intractable epilepsies.