You have no items in your cart.
Fintepla Successfully Managed Patient’s Hard-to-treat Seizures
Fintepla (fenfluramine) successfully managed the severe and difficult-to-treat seizures of a 20-year-old woman with Dravet ...
More from Dravet Syndrome News
-
-
Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds
AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology ... -
Nominations Open for 2022 Eurordis Black Pearl Awards
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. ... -
UK Charity Awards £2.5M to 11 Projects Into Rare Childhood Diseases
Great Ormond Street Hospital (GOSH) Charity and Sparks are investing £2.5 million (about $3.5 million) ... -
Discovery Stabilizes Sodium Channel, May Avert Seizures
Controlling a biological reaction that stabilizes part of the sodium channel in nerve cells can become ... -
Centogene Sets Mission to Cure 100 Rare Diseases in Next 10 Years
At its first virtual investor event, biotech company Centogene set a bold mission: to cure ... -
Survey: Parents, Doctors Satisfied With Epilepsy Teleconsultations
In a survey of physicians and families at a rare pediatric epilepsy center, teleconsultation appointments ... -
Applications Open for Patient Assistance Grants Program
The Dravet Syndrome Foundation (DSF) is accepting applications for its Patient Assistance Grants (PAG) program ... -
EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes
To help patient advocacy leaders and their partners better understand how global health statistics codes ... -
Get Ready for NORD’s Patient and Family Forum, Set for June 26-27
Those who wish to gain practical tools for living optimally with rare diseases are encouraged ... -
Zogenix Opens Photo Contest to Benefit Advocacy Groups
The biopharmaceutical company Zogenix is introducing a Dravet syndrome community photo-sharing campaign and contest to ... -
Rare 2030 Action Campaign Pushing for European Plan for Rare Diseases
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European ... -
Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hill
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the ... -
Supporters Set to ‘Join the Fight’ During Awareness Month
Activities ranging from submitting names for a virtual remembrance wall to swapping out Facebook photo ... -
Takeda Closes Deal With Ovid for Soticlestat
Ovid Therapeutics has closed an agreement under which Takeda Pharmaceuticals will secure global rights to develop ... -
Ataluren Fails to Lower Seizure Frequency in Children in Small Trial
Ataluren, in development by PTC Therapeutics as a possible treatment for Dravet syndrome, showed an ... -
Zogenix Launches Global Program to Boost Access to Fintepla
Zogenix has launched a global access program for Fintepla (fenfluramine) to make the medication available ... -
Model Using Brain Signals May Work as Early Warning System for Seizures
Researchers have developed a mathematical model to identify electrical signal patterns in the brain that ... -
Envision Will Study Dravet Natural History in Children to Learn Impact
The first participant has been enrolled in Envision, a natural history study that will follow ... -
Quick Approval Given COVID Vaccines Raise Concerns, Rare Disease Patients Say
More than half of the people with rare diseases and their caregivers, asked in a ... -
#AANAM – Caregivers Tie Fintepla to Quality-of-life Improvements
Caregivers of children with Dravet syndrome who are taking Fintepla (fenfluramine) report sustained seizure reductions ... -
EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to ... -
COVID-19 Vaccines Safe for Dravet Patients, UK Survey Suggests
Vaccines against COVID-19 appear to be safe and well-tolerated in patients with Dravet syndrome, a ... -
Takeda Buys Global Rights to Soticlestat for Rare Epileptic Disorders
Takeda Pharmaceuticals has acquired the global rights to develop and market the investigational oral therapy soticlestat (OV935/TAK-935) ... -
Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds
As anyone affected by a rare disease knows, treating the illness while trying to go ... -
In 1st Strategic Plan, DSUK Focuses on Research, Awareness, Education
In its 13th year, the nonprofit Dravet Syndrome UK (DSUK) — which seeks to improve ... -
Foundation Promoting New Dravet ICD-10 Codes
The Dravet Syndrome Foundation (DSF) is promoting awareness of the new global health statistics codes ... -
Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day
Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is ... -
UK Survey: Risks of Severe Outcomes from COVID-19 Not Increased Significantly
The risk of severe COVID-19 outcomes does not appear to increase in people with Dravet ... -
The Arc Awarded Comcast NBCUniversal Grant for Special Education Initiative
A $200,000 Comcast NBCUniversal grant has been awarded to The Arc to increase access to ... -
Jazz Pharma to Gain Access to Epidiolex in $7.2B Acquisition of GW Pharma
Jazz Pharmaceuticals is set to acquire GW Pharmaceuticals and gain access to a diversified pipeline ... -
Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion
Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of ... -
Rare Disease Day at NIH, Set for March 1, Growing Year by Year
Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place ... -
Proposed Maryland Law Would Create ‘Seizure Safe Schools’
If a Maryland bill called Brynleigh’s Act becomes law, the state will join five others ... -
Eurodis Survey: Healthcare Experience Worse for Rare Disease Patients
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, ... -
Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, ... -
Diacomit Safe, Effective at Easing Seizures in Clinical Use Reaching Out 2 Years
Diacomit (stiripentol) is safe and effective at reducing the frequency of different types of seizures ... -
Pandemic Won’t Stop Rare Disease Day on Feb. 28
Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 ... -
Fintepla Available in Germany as Add-on Seizure Therapy
After its approval in Europe in December, Fintepla (fenuramine) now is commercially available in Germany ... -
International Epilepsy Day Shines Spotlight on Brain Disorder Affecting Millions
International Epilepsy Day (IED), falling on Feb. 8 this year, calls attention to the needs ... -
NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns
While progress was made last year on newborn screening and other policy issues critical to ... -
Genetic Testing Program Lowered Age of Molecular Diagnosis in Children
By implementing a free-of-charge epilepsy genetic testing program, it is possible to lower the average ... -
Stoke’s TANGO Found to Boost Dravet Protein in Mice Brains
The use of TANGO antisense oligonucleotide-based technology, developed by Stoke Therapeutics, resulted in a dose-dependent ... -
NORD’s Caregiver Respite Program Continues Through Pandemic
Caring for a loved one with a rare disease, especially during these uncertain times, demands ... -
Fintepla Did Not Change Growth in Children With Dravet
Fintepla has no substantial impact on growth, in terms of height and weight, in children ... -
NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences ... -
UN Reclassifies Cannabis in Nod to Plant’s Therapeutic Prospects
After some 59 years, the chief drug policy-making entity of the United Nations (UN) has ... -
Tevard, Zogenix Partner to Advance Gene Therapies for Genetic Epilepsies
Tevard Biosciences and Zogenix announced entering a collaboration agreement to advance potential gene therapies for ... -
Oral Fintepla Approved in Europe as Add-On Seizure Therapy
Zogenix’s Fintepla (fenfluramine) has been approved by the European Commission (EC) as an add-on treatment ... -
Epilepsy Genetic Screening Program Can Speed Up Diagnosis of SCN1A-related Diseases
A no-cost genetic screening program, sponsored by Stoke Therapeutics, helped speed up molecular diagnosis of ... -
FDA Allows Higher Dose of STK-001 for Adolescents, Children in MONARCH Trial
The U.S. Food and Drug Administration (FDA) will allow an additional higher dose of STK-001 ... -
UK Survey Examines Effects of COVID-19 on Dravet Patients
While Dravet syndrome patients don’t appear to be at increased risk of severe COVID-19 complications, ... -
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe
The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and ... -
1st Patient Dosed in Phase 2 Study of EPX-100 for Dravet Syndrome
The first patient has been successfully dosed in a Phase 2 clinical trial testing Epigenix ... -
Specific ICD-10 Codes for Dravet Ultimately Could Improve Patient Outcomes
Dravet syndrome now has its own global health statistics codes — known as “ICD-10” codes ... -
Epilepsy Awareness Month Calls Attention to Seizure Disorders, Dravet, LGS
National Epilepsy Awareness Month is underway, with a variety of events and activities aimed at ... -
CHMP Favors Fintepla as Add-on Dravet Treatment Across Europe
Fintepla — low-dose oral fenfluramine — moved one step closer to European approval, following a favorable opinion ... -
UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner
A photo of a bespectacled young boy, his red baseball cap slightly askew as he ... -
Fintepla Led to ‘Profound’ Reduction in Seizure Frequency in Young Dravet Patients, Extension Study Shows
Long-term treatment with Fintepla can reduce the frequency of seizures in children and adolescents with Dravet ... -
First Patient Enrolled in Phase 2 Trial of EPX-100, Potential Add-on Therapy for Dravet
The first patient has been enrolled in Epygenix Therapeutics’ multicenter Phase 2 clinical trial that ... -
Eisai Begins Phase 3 Trial Testing Lorcaserin at Reducing Dravet Seizure Frequency
Following consultation with the U.S. Food and Drug Administration (FDA), Eisai has initiated a Phase ... -
Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos
Same But Different, a nonprofit U.K. group that uses art for social change, is inviting ... -
Dravet Syndrome Foundation Readies for Online ‘Day of Dravet’
Bringing together patients and their families — virtually, this year — the Dravet Syndrome Foundation ... -
Fycompa May Open to Younger Children With Select Seizures in Europe
The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency (EMA), recommended lowering ... -
Fintepla Reduces Seizure Frequency in Dravet Syndrome Youngsters, Pivotal Phase 3 Trial Reports
Fintepla (fenfluramine) oral solution, developed by Zogenix, significantly reduced seizure frequency in children and teenagers ... -
Fintepla Unlikely to Cause Heart Valve Problems, PAH in Young Patients, Extension Study Finds
Children with Dravet syndrome receiving Fintepla (fenfluramine) to control their seizures are unlikely to develop ... -
Soticlestat Significantly Reduces Seizure Frequency in Children, Top-line Data Show
The investigational oral therapy Soticlestat (OV935/TAK-935), developed by Ovid Therapeutics and Takeda Pharmaceuticals, safely and ... -
First Patient Dosed in MONARCH Clinical Trial Testing Stoke’s STK-001
The first patient has been dosed in Stoke Therapeutics‘ Phase 1/2a MONARCH trial, which is investigating ... -
Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed ... -
Epilepsy Foundation’s Community Day is Virtual This Year
The Epilepsy Foundation’s Community Day, being held virtually this year, will offer patients, caregivers, and ... -
EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US
The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to ... -
SCN1A Gene Activation Reduced Seizures, Improved Behavior in Dravet Mice
Activating the SCN1A gene, which is deficient in most patients with Dravet syndrome, reduced febrile ... -
New Streaming Channel Showcases Rare Disease Films
When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated ... -
Epidiolex Now Available in U.S. for Dravet, LGS Patients Age 1 and Older
The U.S. Food and Drug Administration (FDA) has expanded the age range for all Epidiolex ... -
Encoded Therapeutics Raises $135M to Advance Potential Dravet Gene Therapy
Encoded Therapeutics has raised $135 million to support the first human clinical trials for ETX101, its ... -
Epidiolex May Interact With Diacomit But With Depacon Seems OK, Small Trial Finds
Use of cannabidiol-based Epidiolex with two anti-epileptic treatments do not appear to raise major safety concerns, ... -
Fintepla Approval ‘Fills Us With a Lot of Pride,’ Zogenix CEO Says in Interview
The recent approval of Fintepla (fenfluramine) was a long time coming for its developer, Zogenix, ... -
National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease ... -
UK Eases Restrictions on Epidyolex for Treating Epileptic Disorders
The United Kingdom Home Office has changed the classification of GW Pharmaceuticals‘ Epidyolex — a treatment for ... -
Enrollment Opens in MONARCH Trial Testing STK-001 in Young Patients
Stoke Therapeutics has begun enrollment for its Phase 1/2a MONARCH study, investigating the safety, tolerability, ... -
Fintepla Earns FDA Approval for Treatment of Seizures in Dravet Syndrome
Note: This story was updated June 26, 2020, to note that the high dose of ... -
Epilepsy Surgery May Not Be Advised in Dravet With SCN1A Mutations, Study Finds
Epilepsy surgery may be ineffective, and even detrimental, in people with Dravet syndrome caused by ... -
CBD May Have Its Own Anti-epileptic Properties, Regardless of Clobazam Use
Add-on treatment with Epidiolex — an oral form of cannabidiol (CBD) — effectively drops the ... -
Coalition Will Address Racial Disparities in Rare Disease Communities
The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on ... -
‘Shine Forward with Dravet’ Is Online Support for Families of Patients
Pharmaceutical company Zogenix has launched “Shine Forward with Dravet,” an online resource to support the ... -
Dravet Syndrome Awareness Month is Here … (Online)
In light of the COVID-19 pandemic, there are several ways to get involved virtually to ... -
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for ... -
Enrollment to Start Later This Year in MONARCH Trial Testing STK-001 for Dravet
Stoke Therapeutics plans to begin enrollment later this year of Dravet syndrome patients for part ... -
Video Games Connect Chronically Ill Children Isolated at Home, Hospital
Dara Riva always had a rule that her 10-year-old son could play video games only ... -
Gene Therapy Shows Promise in Mouse Model of Dravet Syndrome
A gene therapy designed to increase the production of a subunit of the defective sodium ... -
Body Cooling May Shorten Refractory Seizures in Dravet and Other Epilepsies
Used in addition to standard treatments, therapeutic hypothermia — based on lowering the body’s temperature ... -
Epidiolex Lowered Premature Mortality in Dravet Mice, Studies Find
Epidiolex, the first plant-derived cannabidiol (CBD) medication to be approved by the U.S. Food and Drug ... -
Heart Abnormalities Unlikely Behind Sudden Unexpected Deaths in Dravet, Study Says
Heart abnormalities are unlikely to be the reason behind the high rate of Sudden Unexpected ... -
Artificial Intelligence May Speed up Epilepsy Diagnoses, Study Suggests
A newly developed artificial intelligence (AI) system could help expedite the diagnosis of epileptic conditions ... -
Fintepla’s Potential Dual Mechanism of Action May Explain Effectiveness at Reducing Seizures in Dravet Syndrome
In addition to promoting the release of serotonin, a brain chemical typically associated with feelings ... -
Epidiolex Now Available in the US as a Non-controlled Substance
In a move that will give Dravet syndrome patients easier treatment access, the U.S. Drug Enforcement ... -
Epidiolex Still Effective at Half the Highest Approved Dose for Reducing Frequency of Convulsive Seizures in Children with Dravet
Epidiolex (cannabidiol), as an add on-therapy administered at either a low or high dose, leads ... -
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia ... -
ELEKTRA Trial Completes Enrollment to Test Soticlestat in Children With Dravet and LGS
The ELEKTRA clinical trial assessing the investigational oral therapy soticlestat (OV935/TAK-935) in children with Dravet syndrome ... -
Antisense Oligonucleotide Treatment Delays Seizures in Dravet Animal Study
An antisense oligonucleotide therapy that lowers the amount of Scn8a transcripts — RNA molecules used as ... -
Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
The number of treatments for children with rare diseases has grown over the past decade, according ... -
Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign ... -
Molecule Seen to Lessen Epileptic Activity, Improve Learning in Early Study
A new small molecule was seen to stimulate certain brain receptors, called NMDA receptors, and ... -
Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles ... -
FDA Extends Review of Fintepla for Dravet Syndrome; Response Expected in June
The U.S. Food and Drug Administration (FDA) extended by three months the review period for ... -
Fintepla Reduces Seizure Frequency in Dravet Children Who Respond Poorly to Diacomit Combo Regimens, Phase 3 Trial Shows
Fintepla (fenfluramine) reduced the frequency of convulsive seizures in children and adolescents with Dravet syndrome ...