Genetic causes behind Huntington’s disease may overlap with those behind rheumatoid arthritis, an unexpected finding that could potentially open the door for new therapeutics targeting both diseases, a study suggests. The study, “Comprehensive epigenetic landscape of rheumatoid arthritis fibroblast-like synoviocytes,” was published in the journal Nature Communications. Rheumatoid…
Should scientists have the right to edit the genes of future generations to eliminate hundreds, if not thousands, of potential rare diseases? Or should researchers restrict their use of genome editing to somatic cells, so that they don’t pass changes on to the next generation? What about stem-cell research, three-parent…
Apathy is linked to physical, cognitive, and behavioral impairments in patients with Huntington’s disease, a study shows. The study, “Relationships Among Apathy, Health-Related Quality of Life, and Function in Huntington’s Disease,” was published in The Journal of Neuropsychiatry and Clinical Neurosciences. Huntington’s disease is characterized by…
Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…
Increasing evidence links a specific fat to Huntington’s disease, and it “merits more attention” as a “high priority” target for potential disease treatments, a review study reports. The study, “The S1P Axis: New Exciting Route for Treating Huntington’s Disease,” was published in the journal Trends in Pharmacological Sciences.
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),…
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