Dravet Syndrome News

September 14, 2021September 14, 2021

New Institute Aims to Leave No Rare Disease Patient Behind

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest ... Read more

September 7, 2021September 7, 2021

Group Focuses on Rare Disease Clinical Trial Participation

Dravet Syndrome News

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running ... Read more

August 31, 2021August 31, 2021

‘Running for Rare’: NORD a Charity Partner of 2021 NYC Marathon

Dravet Syndrome News

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both ... Read more

August 24, 2021August 24, 2021

‘Not Alone in the Dark’: New Guide for, by Caregivers of Children With Rare Diseases

Dravet Syndrome News

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The ... Read more

August 24, 2021August 24, 2021

Fintepla Photo Contest Raises $50K for Dravet, Epilepsy Foundations

Dravet Syndrome News

A total of $50,000 has been raised though a Zogenix photo contest to support the Dravet Syndrome Foundation (DSF) and the Epilepsy Foundation. The company’s donation, to be equally divided, was ... Read more

August 17, 2021August 17, 2021

Register Now for Global Genes’ RARE Patient Advocacy Summit

Dravet Syndrome News

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending ... Read more

August 17, 2021August 17, 2021

New Program Supports Siblings of Rare Epilepsy Patients

Dravet Syndrome News

Siblings of children with Dravet syndrome often grow up “in an environment permeated by stress, anxiety, and fear for their loved one’s well-being,” according to the biopharmaceutical company Zogenix. Now, a ... Read more

August 10, 2021August 10, 2021

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

Dravet Syndrome News

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for ... Read more

August 3, 2021August 3, 2021

CAMP4 Buys Global Rights to OPKO Health’s RNA-based Therapeutics

Dravet Syndrome News

CAMP4 Therapeutics has entered an exclusive worldwide licensing agreement to develop, manufacture, and commercialize OPKO Health’s RNA-based technology as potential disease-modifying treatments for Dravet syndrome. As part of the deal, ... Read more

July 27, 2021July 27, 2021

US Patient Groups Give Thumbs-Up to Rule Against Surprise Billing

Dravet Syndrome News

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other ... Read more

July 20, 2021July 20, 2021

Fintepla Successfully Managed Patient’s Hard-to-treat Seizures

Dravet Syndrome News

Fintepla (fenfluramine) successfully managed the severe and difficult-to-treat seizures of a 20-year-old woman with Dravet syndrome, demonstrating the medication’s potential in real-life for treating such challenging cases, according to researchers. ... Read more

July 20, 2021July 20, 2021

Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds

Dravet Syndrome News

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies ... Read more

July 13, 2021July 13, 2021

Nominations Open for 2022 Eurordis Black Pearl Awards

Dravet Syndrome News

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work ... Read more

July 13, 2021July 13, 2021

UK Charity Awards £2.5M to 11 Projects Into Rare Childhood Diseases

Dravet Syndrome News

Great Ormond Street Hospital (GOSH) Charity and Sparks are investing £2.5 million (about $3.5 million) in 11 medical research projects that will investigate rare childhood diseases, including Dravet syndrome. The ... Read more

July 6, 2021July 6, 2021

Discovery Stabilizes Sodium Channel, May Avert Seizures

Dravet Syndrome News

Controlling a biological reaction that stabilizes part of the sodium channel in nerve cells can become a new approach to prevent epileptic seizures, according to a mouse study. This reaction, called ... Read more