Fintepla RTF Update

By |2019-07-15T16:38:30-05:00July 16th, 2019|Categories: Research / Treatments|

Zogenix To Resubmit New Drug Application Zogenix, Inc. anticipates that they will be resubmitting their New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) in the third quarter, 2019 without additional chronic toxicity studies for Fintepla (ZX008, low dose fenfluramine). In April, the FDA issued a Refusal to File (RTF) [...]

$104M to Gene Therapy

By |2019-07-15T20:31:01-05:00July 16th, 2019|Categories: Research / Treatments|

Encoded Therapeutics Finances $104 Million to Advance Lead Gene Therapy Program Encoded Therapeutics, Inc. announced they will use $104 million in Series C financing to advance its lead gene therapy program in Dravet syndrome and its preclinical pipeline. They also unveiled their precision gene therapy platform. Encoded presented preclinical data on their approach [...]

Stoke BUTTERFLY Study

By |2019-06-13T21:22:27-05:00June 15th, 2019|Categories: Research / Treatments|

Stoke BUTTERFLY Study The BUTTERFLY Study, conducted by Stoke Therapeutics, is collecting information about the mental development, behavior, movement, communication skills, seizure frequency, and sleep pattern of young people with Dravet syndrome. The BUTTERFLY Study is an observational study, which means that no investigational medications or other treatments will be provided. Participants will [...]

Nayzilam Nasal Spray

By |2019-06-13T16:56:59-05:00June 15th, 2019|Categories: Research / Treatments|

FDA approves Nayzilam Nasal Spray The FDA has approved Nayzilam(®)(midazolam) Nasal Spray for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e. seizure clusters, acute repetitive seizures). The nasal spray is designed as a single-use treatment for patients 12 years and older. It can be carried with a patient and [...]

Free Testing

By |2019-05-15T10:18:57-05:00May 15th, 2019|Categories: Research / Treatments|

Behind the Seizures Expansion BioMarin, Invitae, Stoke Therapeutics, and Xenon Pharmaceuticals recently announced the expansion of Behind the Seizures. Previously available to patients ages 2-4 years, the program has expanded to offers Invitae's comprehensive epilepsy panel at no cost to any child up to 60 months who has had an unprovoked seizure. More [...]

Seizure Burden

By |2019-05-13T13:51:17-05:00May 15th, 2019|Categories: Research / Treatments|

Seizure burden in severe early-life epilepsy: Perspectives from parents DSF is pleased to share a recent publication by Anne Berg, PhD: Seizure burden in severe early-life epilepsy: Perspectives from parents. Dr. Berg is a Research Professor of Pediatrics (Neurology and Epilepsy) at Ann and Robert H Lurie Children's Hospital of Chicago with over [...]

Stiripentol Access

By |2019-05-15T10:20:51-05:00April 15th, 2019|Categories: Research / Treatments|

Accessing Stiripentol in the US DSF received notice on the process for patients in the US to secure Diacomit (Stiripentol), effective April 29th. Should patients need Diacomit® before April 29, 2019, they should continue with their current method for medication access, otherwise please refer to the following information:  As of April 29, 2019, [...]

Fintepla RTF

By |2019-05-13T14:02:21-05:00April 15th, 2019|Categories: Research / Treatments|

Zogenix Receives Refusal to File The U.S. Food & Drug Administration (FDA) has issued a Refusal to File (RTF) letter to Zogenix, Inc. for the rolling submission of their New Drug Application (NDA) for Fintepla (ZX008, low-dose fenfluramine). The FDA cited two deficiencies and is asking for additional non-clinical animal study data and [...]

Behavior Survey

By |2019-05-13T14:04:04-05:00April 15th, 2019|Categories: Research / Treatments|

Caregiver Survey: Understanding Behavior DSF is proud to partner with the Child Neurology Foundation to conduct a survey to understand caregivers’ experiences managing disruptive or harmful behavior in children with neurologic conditions, such as Dravet syndrome. Results will be shared with health care providers, so they are better able to serve children with [...]

Fintepla NDA

By |2019-05-13T14:10:44-05:00March 15th, 2019|Categories: Research / Treatments|

Zogenix Submits New Drug Applications Zogenix, Inc. announced it has completed its rolling submission of a New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) and submitted a Marketing Authorization Application (MMA) to the European Medicines Agency (EMA) for FINTEPLA (ZX008, low-dose fenfluramine). Both applications are based on data from [...]