Updates

Just Published, News Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
News Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
News Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
News Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29
News Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar
News Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
News FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar
News With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
News Epidiolex-Clobazam Interaction May Explain Reductions in Seizures, Study Suggests
News HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases
News Ella the Jellyfish Becomes First Amazon Alexa Skill to Offer Fun and Support for LGS Children
News NICE Favors Adding Epidyolex, Cannabidiol Oral Solution, to NHS for Lennox-Gastaut Patients in England
News Rare Disease Film Festival Highlights Patient and Researcher Unity
News Onfi as Add-on Lennox-Gastaut Therapy Lowers Drop Seizure Rates, Study Reports
News Enrollment Completed in Phase 3 Trial Testing Fintepla to Treat Seizures in Lennox-Gastaut Syndrome Patients
Just Published, News Long-term Treatment With Soticlestat Induces Sustained Reduction in Seizure Frequency in Rare Epilepsies, Initial Data Show
News Epidyolex, Oral CBD, Approved in Europe to Treat Lennox-Gastaut and Dravet Syndrome Starting at Age 2

April 2, 2020April 2, 2020
Epidiolex More Effective than Artisanal Cannabidiol for Controlling Seizures, Early Study Suggests

Oral treatment with pharmaceutical cannabidiol (CBD), sold under the brand Epidiolex, seems to be more effective to control seizures than artisanal preparations of CBD, ... Read more
April 2, 2020April 2, 2020
NORD’s Advice on COVID-19: You’re Stronger Than You Think

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to ... Read more
March 26, 2020March 26, 2020
Spontaneous Mutations Linked to LGS in 2 Boys, Study Reports

Spontaneous mutations affecting a critical protein produced in nerve and muscle cells, called EEF1A2, were found to be the underlying cause of Lennox-Gastaut syndrome (LGS) ... Read more
March 26, 2020March 26, 2020
NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because ... Read more
March 23, 2020March 23, 2020
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 Monaco, PhD, of Italy remains confident that the Paris-based ... Read more

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ELEKTRA Trial Testing Soticlestat Completes Early Enrollment of Children With LGS, Dravet
March 19, 2020March 19, 2020
The ELEKTRA trial testing the investigational oral therapy soticlestat (OV935/TAK-935) in children with Lennox-Gastaut syndrome (LGS) ...
News

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
March 12, 2020March 12, 2020
The number of treatments for children with rare diseases has grown over the past decade, ...
uncategorized

Anti-seizure Therapy Banzel Safe, Effective in Children, Adolescents with LGS, Review Finds
March 12, 2020March 12, 2020
Banzel (rufinamide) — an approved add-on, anti-seizure oral therapy — is safe and effective ...
News

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
March 5, 2020March 5, 2020
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media ...
uncategorized

LGS Patients with Mitochondrial Dysfunction May Benefit from Keto, Small Study Suggests
March 5, 2020March 5, 2020
Following a ketogenic (high-fat, low-carb) diet may help reduce seizures and improve cognitive function ...
News

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29
March 3, 2020March 3, 2020
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review ...

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Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar
February 28, 2020February 28, 2020
Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders ...
Fintepla Cuts Lennox-Gastaut Drop Seizure Frequency, Trial Shows
February 27, 2020February 27, 2020
Fintepla (ZX008), an investigational therapy from Zogenix, significantly lowered the frequency of drop seizures ...
Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
February 20, 2020March 3, 2020
Researchers Pinpoint Novel Genetic Variant as the Cause for LGS and Mild Intellectual Impairments in Korean Family
February 13, 2020February 13, 2020
FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar
February 11, 2020February 11, 2020
With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
February 7, 2020February 7, 2020

Distinct Mutations in Calcium Channel Gene May Lead to LGS, Study Suggests
February 6, 2020February 6, 2020
Distinct mutations in the CACNA1A gene — which contains instructions for producing a subunit ...
Christianson Syndrome Unexpectedly Identified as LGS Cause in Two Children, Case Study Reports
January 30, 2020January 30, 2020
FDA Approves Valtoco Nasal Spray for Treating Seizure Clusters
January 23, 2020January 23, 2020
Fycompa as Add-on Treatment Helps Lower Seizures in LGS Patients, Study Suggests
January 16, 2020January 16, 2020

People With Late-Onset LGS May Maintain Their Cognitive and Functional Abilities, Study Finds
January 9, 2020January 9, 2020
People who start showing signs of Lennox-Gastaut syndrome later in life seem to be ...
NHS Allows Adding Epidyolex, Cannabidiol Oral Solution, to Treat Lennox-Gastaut Patients in England
January 2, 2020January 2, 2020
CBD Complements Clobazam to Prevent Seizures in Distinct Ways, Early Study Suggests
December 19, 2019December 19, 2019

News

Epidiolex-Clobazam Interaction May Explain Reductions in Seizures, Study Suggests
December 12, 2019December 12, 2019
Epidiolex-associated reduction in the frequency of drop seizures may be caused by an interaction between its ...
News

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases
December 6, 2019
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or ...
News

Ella the Jellyfish Becomes First Amazon Alexa Skill to Offer Fun and Support for LGS Children
December 5, 2019December 5, 2019
Children with Lennox-Gastaut syndrome, and their families and caregivers can now access the first ...
News

NICE Favors Adding Epidyolex, Cannabidiol Oral Solution, to NHS for Lennox-Gastaut Patients in England
November 21, 2019November 21, 2019
The National Institute of Health and Care Excellence (NICE), an arm of the United Kingdom’s ...
News

Rare Disease Film Festival Highlights Patient and Researcher Unity
November 14, 2019
Rare disease-themed videos glowed on a large screen before an audience of people in ...
News

Onfi as Add-on Lennox-Gastaut Therapy Lowers Drop Seizure Rates, Study Reports
November 14, 2019November 14, 2019
Onfi (clobazam), as an adjunct seizure treatment for Lennox-Gastaut syndrome (LGS), significantly reduced the number ...
News

Enrollment Completed in Phase 3 Trial Testing Fintepla to Treat Seizures in Lennox-Gastaut Syndrome Patients
November 7, 2019November 14, 2019
Zogenix has enrolled the last patient in a Phase 3 clinical trial evaluating its ...
Just Published, News

Long-term Treatment With Soticlestat Induces Sustained Reduction in Seizure Frequency in Rare Epilepsies, Initial Data Show
October 31, 2019November 14, 2019
Long-term treatment with the investigational oral therapy soticlestat (OV935/TAK-935) can reduce the median seizure frequency ...
News

Epidyolex, Oral CBD, Approved in Europe to Treat Lennox-Gastaut and Dravet Syndrome Starting at Age 2
October 24, 2019October 25, 2019
The European Commission has approved Epidyolex (cannabidiol, CBD) as an add-on therapy to clobazam to treat ...

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