ENDYMION Trial Update

By |2019-10-16T14:42:19-05:00October 16th, 2019|Categories: Research / Treatments|

ENYDMION Trial Reports Positive Initial Data Ovid Therapeutics recently announced positive initial data from the ENDYMION trial, Phase 2 open-label extension study of soticlestat (OV935/TAK935) in patients with rare developmental and epileptic encephalopathies (DEE) such as Dravet syndrome and Lennox-Gastaut syndrome (LGS). Soticlestate is a potent, highly-selective, first-in-class inhibitor of the enzyme cholesterol [...]

Fintepla NDA Resubmitted

By |2019-10-16T14:43:00-05:00October 16th, 2019|Categories: Research / Treatments|

Zogenix Resubmits New Drug Application Zogenix, Inc. recently announced that it has resubmitted its New Drug Application (NDA) for FINTEPLA® (ZX008, fenfluramine) for the treatment of seizures associated with Dravet syndrome to the U.S. Food and Drug Administration (FDA). The NDA is based on data from two pivotal Phase 3 trials and an [...]

Breastfeeding Research Survey

By |2019-09-13T11:45:31-05:00September 15th, 2019|Categories: Research / Treatments|

Breastfeeding Research Survey Dr. Douglas Nordli and colleagues at The University of Chicago are conducting a study on Breastfeeding and its impact in Dravet syndrome. This research project aims to find the association between breastfeeding practices and age of onset, severity, morbidity and mortality in patients with Dravet syndrome. This non-interventional, anonymous study [...]

Epygenix IND Approval

By |2019-08-12T10:44:39-05:00August 15th, 2019|Categories: Research / Treatments|

Epygenix Therapeutics Receives IND Approval Epygenix Therapeutics, Inc., a precision medicine-based biopharmaceutical company,  announced that they have received an Investigational New Drug Application (IND) approval from the U.S. Food and Drug Administration (FDA) for the treatment of patients with Dravet syndrome. EXP-100 is a first-generation antihistamine safely used to treat itch in the [...]

Stoke Orphan Drug

By |2019-08-12T15:28:14-05:00August 15th, 2019|Categories: Research / Treatments|

Stoke Granted Orphan Drug Designation Stoke Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead product candidate, STK-001. STK-001 is an investigational new treatment that has the potential to be the first disease-modifying therapy to address the underlying genetic cause of Dravet syndrome. It [...]

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 [...]