Epilepsy syndromes

Emmes Employees Contribute to New England Journal of Medicine Report on Pregnant Women with Epilepsy

Retrieved on: 
Monday, February 8, 2021

"The research addressed the important issue of how to care for women with epilepsy during pregnancy and postpartum," said Dr. Wilson, who leads Emmes' neurology and mental health research unit.

Key Points: 
  • "The research addressed the important issue of how to care for women with epilepsy during pregnancy and postpartum," said Dr. Wilson, who leads Emmes' neurology and mental health research unit.
  • "It is one of the first studies to compare seizure frequency of pregnant women with epilepsy with a non-pregnant control group.
  • One conclusion of the study was that the percentage of women with epilepsy with a higher incidence of seizures during pregnancy was similar to that in women with epilepsy who were not pregnant.
  • The study findings suggest pregnancy may not make seizures worse among women with epilepsy whose seizure medication is carefully managed.

Taysha Gene Therapies Receives Rare Pediatric Disease and Orphan Drug Designations for TSHA-105 for the Treatment of Epilepsy Caused by SLC13A5 Deficiency

Retrieved on: 
Tuesday, January 19, 2021

There are no approved therapies for epilepsy caused by SLC13A5 that address the underlying cause of this disease, said RA Session II, President, Founder and CEO of Taysha.

Key Points: 
  • There are no approved therapies for epilepsy caused by SLC13A5 that address the underlying cause of this disease, said RA Session II, President, Founder and CEO of Taysha.
  • SLC13A5 is a form of infantile epilepsy caused by mutations in the SLC13A5 gene.
  • The disorder is an autosomal recessive disorder, so two copies of the mutated gene must be inherited to affect an infant.
  • This rare form of epilepsy manifests as developmental delay, and seizures beginning within the first few days of life.

Stoke Therapeutics Announces Presentations Related to the Company’s Work to Advance STK-001, the First Potential New Medicine to Target the Underlying Cause of Dravet Syndrome at the American Epilepsy Society (AES) 2020 Annual Meeting

Retrieved on: 
Friday, December 4, 2020

Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life.

Key Points: 
  • Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life.
  • Clinical Diagnosis
    22 children and adolescents with Dravet syndrome were enrolled in the Companys BUTTERFLY observational study and included in a baseline analysis.
  • We continue to generate strong preclinical evidence supporting the potential for STK-001 to address the underlying cause of Dravet syndrome and reduce seizure frequency.
  • Our goal is to develop a medicine that goes beyond seizure control to address many of the other comorbidities associated with Dravet syndrome.

Zogenix and Leading Experts to Present New Rare Epilepsy Data at AES 2020

Retrieved on: 
Tuesday, December 1, 2020

Zogenix will also host a virtual scientific exhibition room and sponsor a continuing medical education (CME) symposium during AES 2020.

Key Points: 
  • Zogenix will also host a virtual scientific exhibition room and sponsor a continuing medical education (CME) symposium during AES 2020.
  • Lennox-Gastaut Syndrome (LGS) is a rare and devastating lifelong childhood-onset epilepsy that can arise from multiple different causes.
  • Although rare, it one of the most common forms of genetic epilepsy.
  • FINTEPLA is also in development for the treatment of seizures associated with Lennox-Gastaut syndrome, another rare and devastating childhood-onset epilepsy.

Stoke Therapeutics Announces Presentations Related to the Company’s Work in Dravet Syndrome at the American Epilepsy Society 2020 Virtual Annual Meeting

Retrieved on: 
Wednesday, November 25, 2020

Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life.

Key Points: 
  • Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life.
  • Patients enrolled to date are representative of patients with Dravet syndrome, and data collected indicate that the selected cognition measures are valid and appropriate for use in patients with Dravet syndrome.
  • The primary endpoints are safety, tolerability and pharmacokinetic profile of STK-001 in Dravet syndrome patients.
  • Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP.

Cerebral Therapeutics Announces Enrollment of First Patient in a Multicenter Randomized Controlled Phase 2b Trial in Epilepsy

Retrieved on: 
Tuesday, November 17, 2020

The trial is enrolling adult epilepsy patients with temporal lobe onset, with or without secondary generalized seizures.

Key Points: 
  • The trial is enrolling adult epilepsy patients with temporal lobe onset, with or without secondary generalized seizures.
  • The study found that subjects experienced a mean 77 percent seizure reduction and extended periods of seizure freedom.
  • We are pleased to advance this important new therapy with the initiation of this randomized clinical trial at leading epilepsy centers in Australia.
  • Cerebral Therapeutics, Inc. is a clinical-stage pharmaceutical company dedicated to improving the lives of patients living with severe refractory epilepsy and other neurological diseases.

Zogenix to Participate in the Stifel 2020 Virtual Healthcare Conference

Retrieved on: 
Wednesday, November 11, 2020

Zogenix is a global biopharmaceutical company committed to developing and commercializing therapies with the potential to transform the lives of patients and their families living with rare diseases.

Key Points: 
  • Zogenix is a global biopharmaceutical company committed to developing and commercializing therapies with the potential to transform the lives of patients and their families living with rare diseases.
  • The companys first rare disease therapy, FINTEPLA (fenfluramine) oral solution has been approved by the U.S. FDA and received a positive CHMP opinion in Europe for the treatment of seizures associated with Dravet syndrome, a rare, severe childhood onset epilepsy.
  • The company has two additional late-stage development programs underway: one for FINTEPLA for the treatment of seizures associated with Lennox-Gastaut syndrome, a different rare childhood-onset epilepsy and another for MT1621, an investigational novel substrate enhancement therapy for the treatment of TK2 deficiency, a rare genetic disorder.
  • MT1621 is being developed through Modis Therapeutics, a Zogenix company.

Zogenix Announces Date Change for Third Quarter 2020 Financial Results and Conference Call and Webcast to November 9

Retrieved on: 
Thursday, October 29, 2020

Zogenix is a global biopharmaceutical company committed to developing and commercializing therapies with the potential to transform the lives of patients and their families living with rare diseases.

Key Points: 
  • Zogenix is a global biopharmaceutical company committed to developing and commercializing therapies with the potential to transform the lives of patients and their families living with rare diseases.
  • The companys first rare disease therapy, FINTEPLA (fenfluramine) oral solution has been approved by the U.S. FDA and received a positive CHMP opinion in Europe for the treatment of seizures associated with Dravet syndrome, a rare, severe childhood onset epilepsy.
  • The company has two additional late-stage development programs underway: one for FINTEPLA for the treatment of seizures associated with Lennox-Gastaut syndrome, a different rare childhood-onset epilepsy and another for MT1621, an investigational novel substrate enhancement therapy for the treatment of TK2 deficiency, a rare genetic disorder.
  • MT1621 is being developed through Modis Therapeutics, a Zogenix company.

Lennox-Gastaut Syndrome (LGS) Awareness Day to Kick Off Epilepsy Awareness Month

Retrieved on: 
Thursday, October 29, 2020

On this day, families affected by LGS will come together to increase public awareness of this rare disease and share their stories of hope for the future.

Key Points: 
  • On this day, families affected by LGS will come together to increase public awareness of this rare disease and share their stories of hope for the future.
  • LGS is a severe epilepsy syndrome that starts in very early childhood and leads to abnormal brain development and progressive intellectual disability.
  • This weekend, the LGS Foundation, the leading organization in the world dedicated to Lennox-Gastaut Syndrome, is hosting its signature awareness event for LGS Awareness Day in advance of a month of awareness activities which will occur in Epilepsy Awareness Month.
  • For more information on Lennox-Gastaut Syndrome or LGS Awareness Day, please visit www.lgsfoundation.org
    View original content to download multimedia: http://www.prnewswire.com/news-releases/lennox-gastaut-syndrome-lgs-awar...

Zogenix to Release Third Quarter 2020 Financial Results and Host Conference Call and Webcast on November 10

Retrieved on: 
Monday, October 26, 2020

Zogenix is a global biopharmaceutical company committed to developing and commercializing therapies with the potential to transform the lives of patients and their families living with rare diseases.

Key Points: 
  • Zogenix is a global biopharmaceutical company committed to developing and commercializing therapies with the potential to transform the lives of patients and their families living with rare diseases.
  • The companys first rare disease therapy, FINTEPLA (fenfluramine) oral solution has been approved by the U.S. FDA and received a positive CHMP opinion in Europe for the treatment of seizures associated with Dravet syndrome, a rare, severe childhood onset epilepsy.
  • The company has two additional late-stage development programs underway: one for FINTEPLA for the treatment of seizures associated with Lennox-Gastaut syndrome, a different rare childhood-onset epilepsy and another for MT1621, an investigational novel substrate enhancement therapy for the treatment of TK2 deficiency, a rare genetic disorder.
  • MT1621 is being developed through Modis Therapeutics, a Zogenix company.