Northwestern University scientists have shown a gene involved in neurodegenerative disease also plays a critical role in the proper function of the circadian clock.
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A novel drug may help increase the effectiveness of radiation therapy for the most deadly form of brain cancer, report scientists at Virginia Commonwealth University Massey Cancer Center. In mouse models of human glioblastoma multiforme (GBM), the new drug helped significantly extend survival when used in combination with radiation therapy.
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Led by Dr. Peiyee Lee and Dr. Richard Gatti, researchers at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA have used induced pluripotent stem cells to advance disease-in-a-dish modeling of a rare genetic disorder, ataxia telangiectasia.
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Researchers from Massachusetts General Hospital and Duke University have identified genetic mutations that appear to underlie a rare but devastating syndrome combining reproductive failure with cerebellar ataxia - a lack of muscle coordination - and dementia.
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UCLA researchers led by Drs. Peiyee Lee and Richard Gatti at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have used induced pluripotent stem cells to advance disease-in-a-dish modeling of a rare genetic disorder, Ataxia Telangiectasia.
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National Institutes of Health researchers used the popular anti-wrinkle agent Botox to discover a new and important role for a group of molecules that nerve cells use to quickly send messages.
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ViroPharma Incorporated, an international biopharmaceutical company committed to developing and commercializing innovative products that address unmet medical needs and rare diseases, today announced the results of a Phase 2 study of VP20621 (non-toxigenic Clostridium difficile; NTCD) a novel treatment approach for preventing recurrent C. difficile infections.
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A bizarre twist on the usual way proteins are made may explain mysterious symptoms in the grandparents of some children with mental disabilities.
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An acute dose of alcohol may cause greater impairment in coordination, learning and memory in the elderly than in young people, according to a study by Baylor University.
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A dysfunction of a certain Calcium channel, the so called P/Q-type channel, in neurons of the cerebellum is sufficient to cause different motor diseases as well as a special type of epilepsy.
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Rapid hearing loss in both ears may be a symptom of the rare but always-fatal Creutzfeldt-Jakob Disease and should be considered a reason for clinicians to test for the disorder.
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What is the cost of rare diseases such as Friedreich's Ataxia? By analyzing direct and indirect costs of care research in BioMed Central's open access journal Orphanet Journal of Rare Diseases calculated that conservatively this rare disease costs between £11,000 and £19,000 per person per year.
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A multi-million Euro initiative is bringing together researchers from across the world to develop new diagnostic tools and new treatments for people with rare diseases and to connect research data in this area on a global scale.
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A groundbreaking, international clinical trial of an iron chelation drug, deferiprone, to treat the rare and devastating neurodegenerative disease, pantothenate kinase-associated neurodegeneration (PKAN), began last month at the trial's only North American site, Children's Hospital & Research Center Oakland.
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Edison Pharmaceuticals today announced the initiation of a phase 2B study entitled, "Safety and Efficacy Study of EPI-743 on Visual Function in Patients with Friedreich's Ataxia."
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The EU Joint Programme - Neurodegenerative Disease Research (JPND) is inviting calls for proposals from research teams across Europe to increase understanding of the factors that put people at risk of developing neurodegenerative diseases (ND) such as Alzheimer's and also to evaluate health and social care strategies for people living with these debilitating illnesses.
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Gulf War veterans with self-reported symptoms of autonomic dysfunction have objective evidence of underlying damage to central and peripheral cholinergic function, a study shows.
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Scientists from the Florida campus of The Scripps Research Institute (TSRI) have uncovered a group of what have been considered relatively minor regulators in the body that band together to suppress the spread of cancer from its primary site.
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UCB, a global biopharmaceutical company focusing on CNS and immunology treatment and research, is sponsoring key sets of VIMPAT® (lacosamide) C-V data at the 66th Annual Meeting of the American Epilepsy Society (AES) in San Diego, Calif., November 30 - December 4.
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Cavalier King Charles with neuropathic pain induced by a condition characterized by a brain–skull size mismatch do not show significant improvement after nonsurgical treatment for the condition, indicate UK study results.
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