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Download the new BCAS Brochure [PDF format]
A stranger put her hands on James Downie's car and begged him not to drive. She wrongly thought his slurred speech and unsteady gait were the result of excess alcohol. "She said 'you can't drive in that condition'," said James. "I showed her my disabled badge and said 'what? My disabled condition?' and she felt very bad about it." James, 29, has a progressive neurological condition called ataxia.
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Using clever chemistry, a Scripps Research team has pinpointed the enzyme target of a drug group that stops the progression of the devastating disease Friedreich's ataxia in mice and may do the same for humans. The findings, developed in collaboration with scientists from Repligen Corporation, help advance this treatment approach one step closer toward human clinical trials, which will be a welcome event for disease sufferers who currently have few treatment options.
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Holly LeBlanc of Holland has lived her life for so long as someone with Friedreich’s ataxia, someone with a degenerative disease that has taken her ability to walk, among other things, she doesn’t think of her life in terms of its daily struggles. “When there’s no treatment or cure, what do you do with it?” she said. “I really just ‘do life.’”Starting a clinical trial this week with the smoking-cessation-drug Chantix, she said, “The biggest word in my mind is hope. Oh my word, there is hope.”
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Scripps Research scientists have successfully created live mice from mouse skin cells, without using embryonic stem cells or cloning techniques that require eggs. This milestone opens the door to the development of exciting therapies, such as using a patient's own cells to grow replacement organs.
The research team, led by Assistant Professor Kristin Baldwin in collaboration with the Mouse Genetics Core, created mice from what are known as induced pluripotent (iPS) cells – stem cells created by reprogramming normal cells taken non-destructively from living animals. While for several years no research team had been able to generate live adult animals from iPS cell lines, the study is part of a trio of new papers showing the feat is possible. Two Chinese groups reported similar results within days of the Scripps Research team's published findings. Each group used different methods, with the Scripps Research team's protocols offering more successful results by some measures. "Reprogramming by iPS cell technology is one of the most exciting areas of research right now," says Baldwin, "because these experiments challenge fundamental paradigms in basic biology and, at the same time, contribute to a technology that offers enormous potential for therapeutic advances."
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ScienceDaily (Sep. 21, 2009) — New data on signalling proteins, called G proteins, may prove important in fighting diseases such as cardiovascular, neurodegenerative disorders, and cancer. For many decades scientists have puzzled on, "How signalling proteins transport and organize in specific areas of the cell?" Researchers from Nano-Science Center and Department of Neuroscience and Pharmacology provide yet unrecognized clues to solve this mystery.
"We now begin to understand how signalling proteins recognize and transport to certain areas of the cell and get a more clear insight on the mechanism of major cellular processes such as cell signalling and growth. This valuable knowledge could be used in the future to understand and cure disease such as depression and Alzheimer's" explains Associate Professor Dimitrios Stamou, Nano-Science Center and Department of Neuroscience and Pharmacology, who led the work.
Cells depend critically on their ability to selectively, transport and isolate proteins in specific areas. Earlier ideas that proposed proteins to move around in the cell by recognizing nanoscale patches in their surrounding membrane, also called lipid rafts, are currently under intense debate. However researchers from Nano-Science Center found a new unsuspected mechanism based on the shape of the membrane and just had their results published in the prominent scientific journal Nature Chemical Biology.
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To evaluate the effectiveness of repetitive locomotor training using a newly developed electromechanical gait device compared with treadmill training/gait training with respect to patient’s ambulatory motor outcome, necessary personnel resources, and discomfort experienced by therapists and patients.
Randomized, controlled, cross-over trial. Sixteen non-ambulatory patients after stroke, severe brain or spinal cord injury sequentially received 2 kinds of gait training. Study intervention A: 20 treatments of locomotor training with an electromechanical gait device; control intervention B: 20 treatments of locomotor training with treadmill or task-oriented gait training. The primary variable was walking ability (Functional Ambulation Category). Secondary variables included gait velocity, Motricity-Index, Rivermead-Mobility-Index, number of therapists needed, and discomfort and effort of patients and therapists during training.
Gait ability and the other motor outcome related parameters improved for all patients, but without significant difference between intervention types. However, during intervention A, significantly fewer therapists were needed, and they reported less discomfort and a lower level of effort during training sessions.
Locomotor training with or without an electromechanical gait trainer leads to improved gait ability; however, using the electromechanical gait trainer requires less therapeutic assistance, and therapist discomfort is reduced.
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