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…
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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…
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Link Between Exact Mutation and Huntington’s Progression May Aid in Early Prognosis, Report Suggests
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…
Vaccinex is hosting a business opinion leaders luncheon on Huntington’s disease in New York City today. As part of the program, the company will discuss its Huntington’s therapy VX15/2503 (VX15). The antibody counters semaphoring 4D (SEMA4D), a molecule that regulates the activation and migration of inflammatory cells in the brain. Scientists believe…
Exercise and physical activity may benefit patients with Huntington’s disease (HD), a new review study shows. The study, “Physical Therapy and Exercise Interventions in Huntington’s Disease: A Mixed Methods Systematic Review,” appeared in the Journal of Huntington’s Disease. Significant research shows that exercise and physical therapy improve the…
This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 million Americans. The campaign kicks off Feb. 1 and culminates with Rare Disease Day on Feb. 28. Organized by…
Neurofeedback training may increase brain connectivity in patients with Huntington’s disease, possibly improving behavior and movement abnormalities, say British researchers at University College London. Their study, “Stimulating neural plasticity with real-time fMRI neurofeedback in Huntington’s disease: A proof of concept study,” appeared in the journal Human Brain Mapping.
The European Medicines Agency (EMA) has granted orphan medicinal product designation to AMT-130, uniQure’s gene therapy candidate for the treatment of Huntington’s disease. AMT-130 is an experimental gene therapy that is being evaluated in studies designed to support an investigational new drug (IND) application with the U.S.
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