Updates

News, syndicated National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US
cystic fibrosis, News, syndicated Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic
News MicroRNA-based Gene Therapy Found to Improve Survival in Mouse Model
News Coalition Will Address Racial Disparities in Rare Disease Communities
News, syndicated COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
News, syndicated Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe
cystic fibrosis, News, syndicated Video Games Connect Chronically Ill Children Isolated at Home, Hospital
News, syndicated Unity and EU-wide Efforts Focus of Online Rare Disease Meeting
News, syndicated Rare Diseases Clinical Research Network Opens Online Survey on COVID-19
News, syndicated Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say
News, syndicated NORD Opens COVID-19 Financial Aid Program for Rare Disease Community
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

July 10, 2020July 10, 2020
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 Survey to measure the full implications, economic and social, ... Read more
July 10, 2020July 9, 2020
What Is Normal?

On a “normal” morning, the house would be quiet, aside from the soft humming of the appliances and the clicking of meandering dog paws ... Read more
July 9, 2020July 9, 2020
Report of Tongue Lesions Highlights Importance of Dental Check-ups in LGS

Two individuals with Lennox-Gastaut syndrome (LGS) who had masses on their tongues, presumably as a result of tongue biting during seizures, were described in ... Read more
July 2, 2020July 2, 2020
Callostomy Using Lasers Appears to Ably Treat Drug-resistant Seizures in LGS

A minimally invasive surgery using lasers to sever connections between the two halves of the brain can effectively and safely lower seizure frequency in ... Read more
June 25, 2020June 25, 2020
Oral CBD Epidyolex Reclassified as Over-the-counter Drug in UK

The United Kingdom Home Office has reclassified Epidyolex (cannabidiol, or CBD) as a Schedule 5 drug, making it easier to obtain for patients with ... Read more

cystic fibrosis, News, syndicated

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic
June 22, 2020June 22, 2020
The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support ...
News

MicroRNA-based Gene Therapy Found to Improve Survival in Mouse Model
June 11, 2020June 11, 2020
A single gene therapy treatment using microRNAs improved growth and development, and decreased lethal ...
News

Coalition Will Address Racial Disparities in Rare Disease Communities
June 10, 2020June 10, 2020
The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused ...
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Mouse Model of LGS Created Based on Mutation Found in Patient
June 4, 2020June 4, 2020
Mice created with a single mutation found in a person with Lennox-Gastaut syndrome showed ...
News, syndicated

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
May 28, 2020May 28, 2020
While the ongoing COVID-19 pandemic won’t have much of an impact on cash available ...
News, syndicated

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe
May 25, 2020May 25, 2020
European authorities must step up efforts to screen babies for a multitude of genetic ...

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Video Games Connect Chronically Ill Children Isolated at Home, Hospital
May 21, 2020May 21, 2020
Dara Riva always had a rule that her 10-year-old son could play video games ...
Unity and EU-wide Efforts Focus of Online Rare Disease Meeting
May 20, 2020
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first ...
Two New CHD2 Mutations Identified, Linked to LGS by Scientists
May 14, 2020May 14, 2020
Rare Diseases Clinical Research Network Opens Online Survey on COVID-19
May 12, 2020May 12, 2020
Epidiolex Safely Lowers Seizure Frequency in Children, 3-Year Study Data Show
May 7, 2020May 7, 2020
NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts
May 1, 2020

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak
April 29, 2020April 29, 2020
FFF Enterprises and BioNews announced today that both rare and orphan disease advocates are ...
Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say
April 28, 2020April 28, 2020
NORD Opens COVID-19 Financial Aid Program for Rare Disease Community
April 23, 2020April 23, 2020
ET-101 Is Biologically Equivalent to Approved Topiramate Solid Products, Study Shows
April 16, 2020April 16, 2020

Epidiolex No Longer Seen as Controlled Substance by US DEA
April 9, 2020April 9, 2020
The U.S. Drug Enforcement Administration (DEA) has reclassified Epidiolex, an oral cannabidiol solution used to treat ...
Epidiolex More Effective than Artisanal Cannabidiol for Controlling Seizures, Early Study Suggests
April 2, 2020April 2, 2020
NORD’s Advice on COVID-19: You’re Stronger Than You Think
April 2, 2020April 2, 2020

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Spontaneous Mutations Linked to LGS in 2 Boys, Study Reports
March 26, 2020March 26, 2020
Spontaneous mutations affecting a critical protein produced in nerve and muscle cells, called EEF1A2, were ...
uncategorized

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum
March 26, 2020March 26, 2020
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 ...
Just Published, News

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
March 23, 2020March 23, 2020
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate ...
uncategorized

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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