Updates

News, syndicated Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe
News, syndicated Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry
News, syndicated NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits
News, syndicated UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner
News Epidiolex Triggers Lithium Toxicity in Boy With LGS, Case Study Reports
News, syndicated Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say
News, syndicated Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos
News, syndicated Same But Different Photo Contest Celebrates People With Rare Diseases
News, syndicated Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input
News, syndicated EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US
News, syndicated New Streaming Channel Showcases Rare Disease Films
News, syndicated National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US
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

November 26, 2020November 26, 2020
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 anxiety in people with rare diseases, negatively affecting their ... Read more
November 19, 2020November 19, 2020
Epidiolex-treated LGS Boy Has No Seizures, Near-normal EEG Pattern, Case Study Reports

Adding Epidiolex, an oral cannabidiol medication, to other treatments successfully stopped epileptic seizures in a boy with Lennox-Gastaut syndrome (LGS), resulting in nearly normal electrical activity ... Read more
November 19, 2020November 19, 2020
Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and ... Read more
November 13, 2020November 12, 2020
Brian Turns 6!

Brian’s birthday party is on Sunday, and he turns 6 years old! I can’t believe how quickly the time has passed. I remember holding ... Read more
November 12, 2020November 12, 2020
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 calling attention to diseases such as Dravet and Lennox-Gastaut ... Read more

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Inovelon/Banzel Seen to Treat LGS Seizures Without Hindering Cognition
November 12, 2020November 12, 2020
The add-on anti-seizure therapy Inovelon (rufinamide) shows good tolerability, with one year of treatment ...
Columns, Into the Unknown – a Column by Jendi Solka

The Risks LGS Caregivers Take
November 6, 2020November 4, 2020
In the spirit of Halloween and the constant uncertainties in our life, I had ...
News, syndicated

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits
November 5, 2020November 5, 2020
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days ...
Columns, Redefining Normal – a Column by Brian Solka

The World Keeps on Turning, Even in the Midst of Tragedy
October 30, 2020October 28, 2020
When Brian’s seizures returned as Lennox-Gastaut syndrome (LGS) after his initial bout with infantile ...
News, syndicated

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner
October 29, 2020October 28, 2020
A photo of a bespectacled young boy, his red baseball cap slightly askew as ...
Columns, Into the Unknown – a Column by Jendi Solka

LGS Caregivers Need a Little TLC, Too
October 23, 2020October 21, 2020
With Lennox-Gastaut syndrome (LGS), you don’t get lucky for very long. The honeymoon phase ...

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Epidiolex Triggers Lithium Toxicity in Boy With LGS, Case Study Reports
October 22, 2020October 22, 2020
Epidiolex (cannabidiol) treatment led to lithium toxicity in a boy with Lennox-Gastaut syndrome (LGS), demonstrating ...
Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say
October 21, 2020October 20, 2020
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience ...
Australian TGA Approves Epidyolex for Treating Seizures Associated with LGS
October 15, 2020October 15, 2020
Our 5 LGS Must-haves to Help Parent Our Son
October 9, 2020October 21, 2020
Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos
October 8, 2020October 8, 2020
Eton Pharmaceuticals Submits New Drug Application for Topiramate Oral Solution
October 8, 2020October 8, 2020

Epidyolex Approved in Scotland to Treat LGS Patients, 2 and Older
October 1, 2020October 1, 2020
The Scottish Medicines Council (SMC) approved Epidyolex (cannabidiol), combined with the anti-epileptic medicine clobazam, ...
As Brian’s Symptoms Worsen, It’s Getting Harder to Manage
September 25, 2020September 23, 2020
EU Panel Favors Fycompa’s Age Range to Include Children Younger Than 12
September 24, 2020September 24, 2020
First Comes Love, Then Comes Marriage, Then Comes a Baby With LGS
September 18, 2020September 16, 2020

Epidiolex-Valproic Acid Combo Can Lower Platelet Counts in Children With LGS, Study Finds
September 17, 2020September 17, 2020
When used alongside valproic acid, Epidiolex (cannabidiol) can increase the risk of thrombocytopenia in children ...
It’s Been One of Those Days
September 11, 2020September 9, 2020
Soticlestat Safely Lowered Seizure Frequency in Children With LGS, Top-line Data Show
September 10, 2020September 10, 2020

Columns, Into the Unknown – a Column by Jendi Solka

Brian Accomplishes a Learning Goal
September 4, 2020September 4, 2020
It’s been a long five months in quarantine. Teaching virtually while supporting my own ...
News, syndicated

Same But Different Photo Contest Celebrates People With Rare Diseases
September 3, 2020September 3, 2020
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, ...
Columns, Redefining Normal – a Column by Brian Solka

Brian Has His First Day of Kindergarten
August 28, 2020August 29, 2020
On my son Brian’s first day of kindergarten, he woke up nicely, babbling to ...
News, syndicated

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input
August 27, 2020August 27, 2020
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, ...
Columns, Into the Unknown – a Column by Jendi Solka

Sleepless in Florida
August 21, 2020August 20, 2020
To say I am not a morning person is an understatement. Just ask my ...
News, syndicated

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US
August 20, 2020August 20, 2020
The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. ...
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FDA Expands Age Range for Epidiolex for Treating LGS Seizures
August 20, 2020August 20, 2020
The U.S. Food and Drug Administration (FDA) has expanded the age range of patients to ...
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Epilepsy Foundation’s Community Day is Virtual This Year
August 18, 2020August 18, 2020
The Epilepsy Foundation’s Community Day, being held virtually this year, will offer patients, caregivers, ...
Columns, Redefining Normal – a Column by Brian Solka

Finding Strength in the Fog
August 14, 2020August 12, 2020
After we finished breakfast, Brian was freed from his eating chair (a donated wheelchair ...