News

Nerve cells contain proteins that send signals to other cells and proteins that receive neighboring cells’ signals. Researchers at Rensselaer Polytechnic Institute in Troy, New York, are trying to understand how the sending proteins end up on one side of a nerve cell’s membrane and the receiving proteins on the…

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…

The U.S. Food and Drug Administration recently granted orphan drug status to Emerald Health’s EHP-102, an investigative therapy for Huntington’s disease. EHP-102 is a patented cannabigerol (CBG) derivative in development for the treatment of Huntington’s and Parkinson’s disease. CBG is a natural cannabinoid with potent effects that has been…

Nerve cells that mimic the molecular features of Huntington’s disease have been created from patients’ skin cells, researchers said. The study, “Striatal neurons directly converted from Huntington’s disease patient fibroblasts recapitulate age-associated disease phenotypes,” was published in Nature Neuroscience. These engineered nerve cells exhibit many of…

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…

Huntington’s symptoms may be a late manifestation of a disease that begins much earlier in embryonic development, according to a recent study. The research, “Chromosomal instability during neurogenesis in Huntington’s disease,” published in the journal Development, corroborates the idea that a lack of the huntingtin protein — not an…

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…

Wave Life Sciences and Takeda Pharmaceutical Company are teaming up to develop nucleic acid therapies for central nervous system disorders such as Huntington’s disease. The agreement gives Takeda the option of co-developing and co-commercializing Huntington’s therapies and of licensing Alzheimer’s and Parkinson’s treatments. The deal also allows Wave…

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…

A case study has reported an association between an exact genetic mutation and Huntington’s disease prognosis, which may help tailor treatments at early stages of the disease. Huntington’s disease is linked to a mutation in the HTT gene that codes for the huntingtin protein. An accumulation of the…