Author Archives: Patricia Inacio PhD

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Takeda Pharmaceuticals has acquired the global rights to develop and market the investigational oral therapy soticlestat (OV935/TAK-935) for developmental and epileptic encephalopathies, including Dravet syndrome, the company has announced. Soticlestat was originally developed by Takeda and Ovid Therapeutics, after the companies entered into a collaboration in 2017.

The U.S. Food and Drug Administration (FDA) has expanded the age range for all Epidiolex (cannabidiol) indications to include patients ages 1 and older, according to its developer, GW Pharmaceuticals (GW). The therapy previously had been approved in the U.S. and in Europe for the treatment of seizures associated…

Encoded Therapeutics has raised $135 million to support the first human clinical trials for ETX101, its gene therapy for SCN1A-positive Dravet syndrome patients. The funding also will be used for a natural history study to better understand the progression of the disease in people carrying SCN1A mutations, which are…

Epidiolex (cannabidiol), as an add on-therapy administered at either a low or high dose, leads to similar clinically relevant reductions in the frequency of convulsive seizures in children with treatment-resistant Dravet syndrome, Importantly, a lower Epidiolex dose shows a better safety and tolerability profile when compared to the higher dose.

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

The U.S. Food and Drug Administration (FDA) extended by three months the review period for Zogenix’s New Drug Application (NDA) for Fintepla (ZX008), an investigational anti-seizure therapy for patients with Dravet syndrome. The agency now expects to provide a decision by June 25 on whether…

Editor’s note: This story has been updated to add that Zogenix resubmitted a new drug application for Fintepla to the FDA in late September.  Treatment with Fintepla (ZX008) can provide a clinically meaningful and profound reduction in the frequency of seizures in Dravet syndrome patients, according to…