The MitoTarget Project was a project funded by the European Commission, under their FP7 programme. It was funding for a call published in 2007.
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Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, announces today the conclusion of the pan-European MitoTarget Project, the EU funded orphan disease project, with submission of the final report to the EC.
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A team of University of Missouri researchers has found that introducing a missing gene into the central nervous system could help extend the lives of patients with Spinal Muscular Atrophy (SMA) - the leading genetic cause of infantile death in the world.
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Zebrafish, popular as aquarium fish, now have an important place in research labs as a model organism for studying human diseases.
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Cedars-Sinai's Regenerative Medicine Institute has pioneered research on how motor-neuron cell-death occurs in patients with spinal muscular atrophy, offering an important clue in identifying potential medicines to treat this leading genetic cause of death in infants and toddlers.
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An innovative strategy for regenerating skeletal muscle tissue using cells derived from the amniotic fluid is outlined in new research published by scientists at the UCL Institute of Child Health.
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Scientists have confirmed that mutations of a gene are responsible for some cases of a rare, inherited disease that causes progressive muscle degeneration and weakness: spinal muscular atrophy with lower extremity predominance, also known as SMA-LED.
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In an effort to identify the underlying causes of neurological disorders that impair motor functions such as walking and breathing, UCLA researchers have developed a novel system to measure the communication between stem cell-derived motor neurons and muscle cells in a Petri dish.
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Biogen Idec Inc., a global biotechnology leader in the discovery, development, manufacturing and commercialization of innovative therapies, today announced its first quarter 2012 results.
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Termed “preimplantation genetic screening (PGS)” the process involves a full chromosome count of embryos to ensure only the healthiest are implanted. It has a 99 per cent accuracy rate, giving hopeful parents the best chance to conceive and carry a healthy baby to term.
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Repligen Corporation today announced positive results from a Phase 1 study to evaluate the pharmacokinetic (PK) and safety profile of RG3039, a novel small molecule drug candidate for the potential treatment of spinal muscular atrophy (SMA).
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Dr Jianguang Ji, Lund University, and Skåne University Hospital, Malmö, Sweden, and colleagues investigated data from the Swedish Cancer Registry. Researchers analyzed Swedish hospital data from 1969 to 2008. They found 1,510 patients with Huntington's disease. During the study period, 91 of those patients subsequently developed cancer. The authors said that was 53% lower than the levels expected for the general population.
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An abnormally low level of a protein in certain nerve cells is linked to movement problems that characterize the deadly childhood disorder spinal muscular atrophy, new research in animals suggests.
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Repligen Corporation today reported financial results for the quarter and nine-month fiscal year ended December 31, 2011.
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Imagine a "smart pill" containing a biodegradable electronic chip that monitors how your body responds to the medicine, broadcasts the information to your iPhone, which then emails the information to your physician.
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Biogen Idec Inc., a global biotechnology leader in the discovery, development, manufacturing and commercialization of innovative therapies, today announced its full year and fourth quarter 2011 results.
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Within the first days of life, screening tests are performed on all newborns born in the U.S. to identify rare and often life-threatening medical conditions that are not apparent at birth. These newborn screening programs have been operating for decades, permitting doctors to intervene early and improve outcomes in potentially devastating conditions.
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The World Health Organization (WHO) reports that India has recorded no cases of polio for one full year - a significant benchmark for the South Asian nation and an encouraging development for health professionals fighting to eradicate the stubborn disease worldwide.
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BioTime, Inc. today announced that it has elected to market progenitors of muscle stem cells bearing hereditary diseases. BioTime will produce the products from five human embryonic stem (hES) cell lines from Reproductive Genetics Institute (RGI) of Chicago, Illinois.
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In a development that could pave the way for treatment for rare neurological diseases and clues to more common ones, physician-scientists at Albert Einstein College of Medicine of Yeshiva University and Montefiore Medical Center, the University Hospital for Einstein, have secured a grant to establish a clinical site for the Network for Excellence in Neuroscience Clinical Trials.
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