The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is…
News
A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…
Epidiolex (cannabidiol) treatment led to lithium toxicity in a boy with Lennox-Gastaut syndrome (LGS), demonstrating a previously unknown, harmful interaction between these two therapies, a case study reports. Given the lack of information about interactions between…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
The Australian Therapeutic Goods Administration (TGA) has approved Epidyolex (cannabidiol) as a treatment for seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome in patients 2 or older, according to developers GW Pharmaceuticals. In order to make Epidyolex available to Australian patients,…
Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…
Eton Pharmaceuticals has submitted a new drug application (NDA) for topiramate oral solution — an anticonvulsant formerly known as ET-101 — to the U.S. Food and Drug Administration (FDA). The NDA covers three indications: as an add-on therapy for treatment of partial-onset seizures, including…
The Scottish Medicines Council (SMC) approved Epidyolex (cannabidiol), combined with the anti-epileptic medicine clobazam, as a treatment option for children and adults with Lennox-Gastaut or Dravet Syndrome.
A European Medicines Agency (EMA) committee has recommended that the age range of Fycompa (perampanel) be expanded in the European Union (EU) to include children 4 and older with focal-onset seizures and those 7 and older with primary generalized tonic-clonic seizures (PGTCS). In the EU, Fycompa — available in…
When used alongside valproic acid, Epidiolex (cannabidiol) can increase the risk of thrombocytopenia in children and adolescents with drug-resistant forms of epilepsy, such as Lennox-Gastaut syndrome (LGS), a study shows. According to the study authors, young patients with these disorders and receiving this combination of anti-seizure medications…
Recent Posts
- EEG headband tracks LGS kids’ sleep disturbances at home
- Making the best of vacationing while caring for a child with LGS
- Combined nerve stimulation linked to greater seizure reductions in LGS
- Navigating my son’s constantly changing eating habits can be tricky
- Effectiveness data due soon from large trial of LGS seizure treatment