Cockroaches can be pretty stubborn, and sometimes it feels like you can’t get rid of them. These nasty creatures are probably the worst imposters you can ever have, and they love dark corners. Most people feel disgusted just by thinking of them, and others are scared. But, the worst part of dealing with cockroaches is…
Back pain is a serious health issue which affects many people and is most often caused by injuries, improper body postures, improper sitting, and standing. Fortunately, this pain can be dramatically relieved with the help of the following exercises. They are also very helpful in the case of knee and hip pain and will correct…
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, and has the nation’s third-highest rate of uninsured residents — with 13.9% of all Oklahomans lacking health coverage. As if that’s not enough, the Sooner…
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare diseases can fall to competing priorities.…
Severe epilepsy in children with Dravet and Lennox-Gastaut syndrome can be safely treated with a medicinal cannabis oil that includes, at low dose, the intoxicating compound in marijuana known as THC, preliminary results of a Phase 1 clinical study suggest. “What makes these results really exciting is it opens up as a treatment option for kids who…
Encoded Therapeutics will further develop its lead precision gene therapy platform for Dravet syndrome and other severe genetic disorders after a financing round brought in $104 million from several investors. The company’s goal is to receive approval and market this platform, which aims to overcome key limitations of current gene therapy technology. “Our mission is…
A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments for the 95% of such illnesses that currently don’t have one. The European Joint Programme on Rare Diseases (EJP RD), established in January 2019, operates…
A genetic phenomenon called double somatic mosaicism was found for a first time in a girl with Dravet syndrome, a case study reports. The study, “Double somatic mosaicism in a child with Dravet syndrome,” was published in Neurology Genetics. Dravet syndrome, a severe type of epilepsy usually evident during the first year of life, is in about 80%…
Zogenix has announced it will resubmit a New Drug Application (NDA) for Fintepla (ZX008), its investigational anti-seizure therapy for patients with Dravet syndrome, likely in the third quarter of this year. The U.S. Food and Drug Administration (FDA) refused the first NDA for Fintepla in April. In a Refusal to File (RTF) letter, the FDA cited…
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although she had known for years that something was wrong, the news that David would battle a lifelong rare disease devastated the single mother of three, plunging…