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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”…

Treatment with investigational medication VX15/2503 halts disease-related loss of brain volume and improves its metabolic activity in Huntington’s disease patients, according to a Phase 2 clinical study. The research, “Clinical Development of VX15/2503 Anti-Semaphorin 4D Antibody as a Potential Treatment for Huntington’s Disease,” was presented by Elizabeth Evans, PhD,…

The investigative therapy IONIS-HTTRx effectively reduced levels of the protein responsible for Huntington’s disease in early-stage patients, according to the findings of an ongoing Phase 1/2 clinical trial. The results also revealed that IONIS-HTTRx is generally safe and well-tolerated by patients, further demonstrating its potential as an alternative therapeutic option…

CHDI Foundation has teamed up with PTC Therapeutics to try to advance PTC’s work on small-molecule compounds that can reduce the production of huntingtin, the protein responsible for Huntington’s disease. The compounds have shown promise. They decreased the protein in a mouse model carrying the human huntingtin gene. And they…