News

Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in Washington, D.C., helping it to strengthen care available and expand as a “center of excellence” for rare…

A database created at the University of Arizona College of Medicine — Phoenix alerts physicians about medications that can be harmful to epilepsy patients on ketogenic diets. The database identifies high-carbohydrate medications that are not recommended for people on ketogenic diets.

In recognition of Rare Disease Day 2018, Bionews Services — which publishes this website — will attend and report on three relevant conferences in the U.S. dealing with policies and programs of importance to patients and their families. The three are among 50 events in 32 states…

The U.S. Food and Drug Administration has granted breakthrough therapy designation to ZX008 to treat seizures associated with Dravet syndrome. This new status adds to the orphan drug and fast track designations previously granted by the FDA and the European Medicines Agency (EMA) and is intended to support the…

A rock-painting contest in Las Vegas. A fashion show in New York. A 7,000-meter race around the Washington Monument that’ll coincide with a similar #Racefor7 event in Bengaluru and Mumbai, India. From Athens to Atlanta, from San Diego to Sydney, people across the globe will mark World Rare Disease…

GW Pharmaceuticals has published positive results of its Phase 3 trial testing Epidiolex, the company’s cannabis-based oral therapy, in people with treatment-resistent Lennox-Gastaut syndrome (LGS). The results, published in the journal The Lancet, also carry promise for Dravet syndrome, another rare and difficult-to-treat epilepsy, as this therapy is currently…

David Curtis Glebe, a retired 64-year-old public prosecutor now living in Millsboro, Delaware, knows he’s lucky to be alive. In mid-2013, while in Arizona, Glebe was diagnosed with pancreatic neuroendocrine cancer (PNET) — the same disease that killed Apple’s founder and CEO Steve Jobs. After three years of progress…

Zogenix has completed enrolling patients in a second Phase 3 trial evaluating ZX008 as a treatment for Dravet syndrome epilepsy. Standard epilepsy medications are unable to counter Dravet-associated seizures, which can cause cognitive impairment and even death. Doctors usually use anticonvulsant medications for the disease, but patient outcomes remain poor. ZX008…

Incorporating brain activity pattern analysis in chemical compounds screenings for Dravet syndrome and other epilepsies can help predict therapies’ effectiveness and minimize chances for off-target effects. That finding comes from the study “Brain activity patterns in high-throughput electrophysiology screen predict both drug efficacies and side effects,” which was published…

Severe respiratory abnormalities induced by seizures are common in patients with Dravet syndrome, and may increase patients’ risk of sudden unexplained death in epilepsy (SUDEP), according to a recent study. The report, “Severe Peri-Ictal Respiratory Dysfunction is Common in Dravet Syndrome,” was published in The Journal of Clinical…