News tagged with fatal genetic disease
NIH clinical trial begins for treatment of rare, fatal neurological disorder
A clinical trial to evaluate a drug candidate called cyclodextrin as a possible treatment for Niemann-Pick disease type C1 (NPC), a rare and fatal genetic disease, will start today, researchers announced. Scientists from ...
Diseases, Conditions, Syndromes
Jan 23, 2013 |
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Researchers find chemical 'switches' for neurodegenerative diseases
By using a model, researchers at the University of Montreal have identified and "switched off" a chemical chain that causes neurodegenerative diseases such as Huntington's disease, amyotrophic lateral sclerosis and dementia. ...
Neuroscience
Nov 27, 2012 |
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Progeria: First-ever treatment for rare childhood aging disease shows improvement in all trial participants
Results of the first-ever clinical drug trial for children with Progeria, a rare, fatal "rapid-aging" disease, demonstrate the efficacy of a farnesyltransferase inhibitor (FTI), a drug originally developed to treat cancer. ...
Diseases, Conditions, Syndromes
Sep 24, 2012 |
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First controlled clinical trial for Juvenile Batten disease to start
After years of building hope for a treatment, Rochester researchers and clinicians will begin the first controlled clinical trial for Juvenile Batten disease this summer, thanks to $1 million in grants from the Food and Drug ...
Diseases, Conditions, Syndromes
May 31, 2011 |
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