News

February 20, 2020February 20, 2020

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

News

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and ... Read more

February 11, 2020February 11, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

News

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous ... Read more

February 7, 2020February 7, 2020

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

News

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over ... Read more

December 12, 2019December 12, 2019

Epidiolex-Clobazam Interaction May Explain Reductions in Seizures, Study Suggests

News

Epidiolex-associated reduction in the frequency of drop seizures may be caused by an interaction between its active component — cannabidiol (CBD) — and clobazam, a well-known anti-seizure medication, according to a ... Read more

December 6, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

News

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s ... Read more

December 5, 2019December 5, 2019

Ella the Jellyfish Becomes First Amazon Alexa Skill to Offer Fun and Support for LGS Children

News

Children with Lennox-Gastaut syndrome, and their families and caregivers can now access the first Amazon Alexa skill — called Ella the Jellyfish — that’s designed to provide support and ... Read more

November 21, 2019November 21, 2019

NICE Favors Adding Epidyolex, Cannabidiol Oral Solution, to NHS for Lennox-Gastaut Patients in England

News

The National Institute of Health and Care Excellence (NICE), an arm of the United Kingdom’s public health system, favors use of the oral cannabidiol solution Epidyolex as an add-on to clobazam ... Read more

November 14, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity

News

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. ... Read more

November 14, 2019November 14, 2019

Onfi as Add-on Lennox-Gastaut Therapy Lowers Drop Seizure Rates, Study Reports

News

Onfi (clobazam), as an adjunct seizure treatment for Lennox-Gastaut syndrome (LGS), significantly reduced the number of weekly drop seizures and its effects were maintained in some patients for up to ... Read more

November 7, 2019November 14, 2019

Enrollment Completed in Phase 3 Trial Testing Fintepla to Treat Seizures in Lennox-Gastaut Syndrome Patients

News

Zogenix has enrolled the last patient in a Phase 3 clinical trial evaluating its lead investigational candidate Fintepla (ZX008) as an add-on therapy for seizures associated with Lennox‑Gastaut syndrome (LGS), the ... Read more

Pin It on Pinterest