News

At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency of working across the aisle to help the estimated 30 million Americans with rare diseases. Rep. Leonard Lance (R-New Jersey) and Sen. Amy Klobuchar (D-Minnesota) spoke to more…

Levetiracetam (marketed as Keppra), an anti-epileptic therapy, is significantly more effective than phenobarbital medication in infants with epilepsy, a recent study found, which could have implications for patients with Dravet syndrome. In the study, “Comparative Effectiveness of Levetiracetam vs Phenobarbital for Infantile Epilepsy,” published in JAMA Pediatrics, scientists…

Stiripentol combined with two other antiepileptic medicines seems to be an effective strategy to improve long-term seizure frequency in Dravet syndrome patients. The study that contains that finding, “Stiripentol efficacy and safety in Dravet syndrome: a 12-year observational study” was published in the journal Developmental Medicine & Child Neurology.

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…