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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 encephalopathies, according to Phase 2 trial data. Developmental and epileptic encephalopathies, or DEE, is an umbrella term that…
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 in a zebrafish model. The study, “NaV1.1 and NaV1.6 selective compounds reduce the behavior phenotype in a…
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 of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…
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) in patients 2 and older. This decision, which follows the favorable opinion of the…
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 of CBD could lead to legal action. The agency “urges the companies to review all claims made for their…
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: A Novel Antiseizure Medication for the Management of Dravet Syndrome,” was published in the journal…
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 by Stoke Therapeutics, the company that is funding the study. BUTTERFLY is an observational study, which means…
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: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…
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 issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…
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 company now intends to test STK-001 in a Phase 1/2 clinical study in the first half of…
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