October 8, 2019October 8, 2019
Cannabis-based Zygel Helps Reduce Seizures in Childhood Epilepsy Syndromes, Trial Reports
News
Zygel (ZYN002) — a cannabis-based gel formulation developed by Zynerba Pharmaceuticals — can significantly reduce seizures by up to ... Read more
October 1, 2019October 1, 2019
Blocking Specific Sodium Channel May Offer Way of Treating Dravet, Early Study Suggests
News
Selectively blocking NaV1.6 — a sodium channel known to be overactive in some epileptic conditions — may offer a ... Read more
September 26, 2019
NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC
News
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a ... Read more
September 24, 2019September 24, 2019
Oral Cannabidiol Epidyolex Approved in Europe as Add-on Treatment for Dravet, Lennox-Gastaut Syndromes
News
The European Commission has approved Epidyolex (cannabidiol) as an add-on therapy to clobazam (brand names Onfi and Sympazan) for the treatment ... Read more
September 17, 2019
FTC Warns Companies Against Unsubstantiated Advertising of CBD Products for Fibromyalgia
News
The U.S. Federal Trade Commission (FTC) sent warning letters to three companies that sell cannabis-based products containing cannabidiol (CBD), ... Read more
September 10, 2019September 10, 2019
Diacomit is Effective as Add-on Therapy to Reduce Refractory Seizures in Dravet, Review Study Finds
News
Diacomit (stiripentol) is an effective add-on oral therapy to reduce the frequency and duration of seizures in patients with ... Read more
September 3, 2019September 3, 2019
BUTTERFLY Study of Children and Teens Now Enrolling with Goal of Better Understanding Dravet
News
The first patient has been enrolled in the BUTTERFLY study, which aims to characterize features of Dravet syndrome in children ... Read more
August 30, 2019August 30, 2019
‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases
News
Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as ... Read more
August 29, 2019August 28, 2019
At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing
News
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert ... Read more
August 27, 2019August 27, 2019
Stoke Therapeutics’ STK-001 Granted Orphan Drug Designation by FDA
News
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation — allowing various development incentives — to ... Read more
August 20, 2019October 8, 2019
EU Approval of Oral Cannabidiol as Add-On Therapy for Dravet Favored by CHMP
News
The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency (EMA), is recommending approval of GW Pharmaceutical’s oral cannabidiol solution ... Read more
August 20, 2019
Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
News
Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you ... Read more

$Diacomit is Effective as Add-on Therapy to Reduce Refractory Seizures in Dravet, Review Study Finds$

October 8, 2019October 8, 2019

Cannabis-based Zygel Helps Reduce Seizures in Childhood Epilepsy Syndromes, Trial Reports

News

Zygel (ZYN002) — a cannabis-based gel formulation developed by Zynerba Pharmaceuticals — can significantly reduce seizures by up to six months in children and adolescents with developmental and epileptic ... Read more

October 1, 2019October 1, 2019

Blocking Specific Sodium Channel May Offer Way of Treating Dravet, Early Study Suggests

News

Selectively blocking NaV1.6 — a sodium channel known to be overactive in some epileptic conditions — may offer a way of treating people with Dravet syndrome, according to work ... Read more

September 26, 2019

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

News

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board ... Read more

September 24, 2019September 24, 2019

Oral Cannabidiol Epidyolex Approved in Europe as Add-on Treatment for Dravet, Lennox-Gastaut Syndromes

News

The European Commission has approved Epidyolex (cannabidiol) as an add-on therapy to clobazam (brand names Onfi and Sympazan) for the treatment of seizures associated with Dravet syndrome and Lennox‑Gastaut syndrome (LGS) ... Read more

September 17, 2019

FTC Warns Companies Against Unsubstantiated Advertising of CBD Products for Fibromyalgia

News

The U.S. Federal Trade Commission (FTC) sent warning letters to three companies that sell cannabis-based products containing cannabidiol (CBD), cautioning them that making unsubstantiated claims about the health benefits ... Read more

$Diacomit is Effective as Add-on Therapy to Reduce Refractory Seizures in Dravet, Review Study Finds$

September 10, 2019September 10, 2019

Diacomit is Effective as Add-on Therapy to Reduce Refractory Seizures in Dravet, Review Study Finds

News

Diacomit (stiripentol) is an effective add-on oral therapy to reduce the frequency and duration of seizures in patients with Dravet syndrome, a review study has found. The study, “Stiripentol: ... Read more

September 3, 2019September 3, 2019

BUTTERFLY Study of Children and Teens Now Enrolling with Goal of Better Understanding Dravet

News

The first patient has been enrolled in the BUTTERFLY study, which aims to characterize features of Dravet syndrome in children and adolescents. The announcement was made in a press release ... Read more

August 30, 2019August 30, 2019

‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

News

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: ... Read more

August 29, 2019August 28, 2019

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

News

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such ... Read more

August 27, 2019August 27, 2019

Stoke Therapeutics’ STK-001 Granted Orphan Drug Designation by FDA

News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation — allowing various development incentives — to Stoke Therapeutics’ STK-001 for the treatment of Dravet syndrome. The ... Read more