News

Two years after approving it, the 28-member European Union will begin enforcing its General Data Protection Regulation (GDPR) — a tough new law that aims to protect the EU’s 512 million citizens, including rare disease patients, from having their medical records misused, sold, or subject to extortion by hackers, third…

Monensin, an antibiotic widely used in feed for animals like cows that chew their cud, can help protect cells against oxidative stress linked to Huntington’s disease, according to a new mouse study. The biochemical pathway that allows a cellular structure known as the Golgi apparatus to fight this oxidative stress…

AllianceRx Walgreens Prime and Walgreens now have several new limited distribution therapies available for its specialty patients, including Teva Pharmaceuticals’ Austedo (deutetrabenazine) for Huntington’s disease. Austedo was approved in 2017 by the U.S. Food and Drug Administration as a treatment for chorea associated with…

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…

For Huntington’s Disease Awareness Week, the Huntington’s Disease Association in the U.K. has a few suggestions for ways to bring the community together and educate others. Because Huntington’s is a rare disease, few are fully aware of its symptoms and signs. Advocates want to use occasions such as this to educate the…

Catherine Martin’s mother died from Huntington’s disease. So did her grandmother and a number of aunts, uncles, and cousins. Yet the Scottish woman didn’t get tested for this genetic illness — which she had a 50-50 chance of developing — until she was 30 years old. She tested negative.

Targeting the Gpr52 receptor can reduce the levels of mutant huntingtin (HTT) proteins and improve Huntington’s disease symptoms, including walking problems, in animal models, a study suggests. The study, “Targeting Gpr52 lowers mutant HTT levels and rescues Huntington’s disease-associated phenotypes,” was published in the journal Brain. G…

Iron accumulation in the mitochondria of brain cells may drive Huntington’s disease progression, researchers report.  Removing excess iron with an FDA-approved treatment was seen to rescue mitochondrial function and improved motor endurance in a mouse model of the disease. Their study, “Brain mitochondrial iron accumulates in Huntington’s disease, mediates…

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…