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Identifying Factors Contributing to Successful Stem Cell Transplants

Posted: Published on August 2nd, 2014

Washington DC - infoZine - Approximately three-quarters of SCID infants who received transplants survived for at least five years. Infants who received transplants within the first 3.5 months of life had the best outcomes. SCID is caused by defects in genes involved in the development and function of infection-fighting T and B cells. Infants with SCID appear healthy at birth but are highly susceptible to infections. If untreated, SCID is fatal, usually within the first year of life. Development of a newborn screening test has made it possible to detect SCID before symptoms appear. The test was added to the U.S. Department of Health and Human Services' Recommended Uniform Screening Panel for newborns in 2010, but to date, only 21 states have implemented newborn screening for SCID. "The findings from this study highlight the positive impact of treating SCID early in life," said NIAID Director Anthony S. Fauci, M.D. "They also suggest that widespread use of newborn screening tests for SCID is warranted to ensure that infants with this rare syndrome receive life-saving transplants." Stem cell transplantation can fully correct the T-cell and, less consistently, the B-cell deficiencies of SCID infants. To identify factors that contribute to successful transplant outcomes, … Continue reading →

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Fundraiser planned to help Dexter family whose son has rare disease

Posted: Published on August 2nd, 2014

Kim Watkins was in Tennessee just starting a new career when she got a phone call that turned her life upside down and dimmed the bright future she thought her family would have. The phone call came in May and confirmed the familys worst fears. Doctors have found lesions on the brain of Glen and Kim Watkins 8-year-old son, Hayden. The lesions are a symptom of adrenoleukodystrophy (ALD) a rare disease that affects 1 in 20,000 boys. The lesions are eating away at Haydens brain and will cause irreversible brain damage, loss of the boys five senses and possibly death if not caught in time. Hayden has a 1 to 2-year window of opportunity to rectify the situation. It successfully takes a transplant to stop this or in two years he will be reduced to a vegetative state, Kim said. Haydens family had known this day could come since he was born. He had been getting annual MRIs done every October, but since the family was slated to move south, the MRI was bumped up by six months. It was during this recent scan that the lesions were first spotted. The family is on a waiting list to receive a … Continue reading →

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Recent advances in stem cell biology

Posted: Published on August 2nd, 2014

PUBLIC RELEASE DATE: 31-Jul-2014 Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research Advances in stem cell research will provide enormous opportunities for both biological and future clinical applications. Basically, stem cells could replicate any other cells in the body, offering immense hope of curing Alzheimer's disease, repairing damaged spinal cords, treating kidney, liver and lung diseases and making damaged hearts whole. The potential for profit is staggering. Prof. Jinhui Chen from Indiana University in USA considered that this field of research still faces myriad biological, ethical, legal, political, and financial challenges. The eventual resolution of these conflicts will determine the success of the research and potentially the face of medicine in the future. The relevant study has been published in the Neural Regeneration Research (Vol. 9, No. 7, 2014). ### Article: " A brief review of recent advances in stem cell biology " by Jinhui Chen1, Libing Zhou2, Su-yue Pan3 (1 Stark Neuroscience Research Institute and Department of Neurological Surgery, Indiana University School of Medicine, Indianapolis, IN, USA; 2 Guangdong-Hongkong-Macau Institute of CNS Regeneration (GHMICR), Jinan University, Guangzhou, Guangdong Province, China; 3 Department of Neurology, Nanfang Hospital, Southern Medical University, Guangzhou, Guangdong Province, China) Chen JH, Zhou LB, Pan SY. … Continue reading →

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Early Stem Cell Transplant Vital in 'Bubble Boy' Disease

Posted: Published on August 2nd, 2014

By Amy Norton HealthDay Reporter WEDNESDAY, July 30, 2014 (HealthDay News) -- Babies born with so-called "bubble boy" disease can often be cured with a stem cell transplant, regardless of the donor -- but early treatment is critical, a new study finds. Severe combined immunodeficiency (SCID), as the condition is medically known, actually refers to a group of rare genetic disorders that all but eliminate the immune system. That leaves children at high risk of severe infections. The term "bubble boy" became popular after a Texas boy with SCID lived in a plastic bubble to ward off infections. The boy, David Vetter, died in 1984 at the age of 12, after an unsuccessful bone marrow transplant -- an attempt to give him a functioning immune system. Today, children with SCID have a high chance of survival if they receive an early stem cell transplant, researchers report in the July 31 issue of the New England Journal of Medicine. In the best-case scenario, a child would get stem cells -- the blood-forming cells within bone marrow -- from a sibling who is a perfect match for certain immune-system genes. But that's not always an option, partly because kids with SCID are … Continue reading →

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Fort Frances Safeway Partners with Muscular Dystrophy Canada to "Make Muscles Move"

Posted: Published on August 2nd, 2014

FORT FRANCES, ONTARIO--(Marketwired - Aug. 1, 2014) - Muscular Dystrophy Canada and Safeway have launched their campaign to "Make Muscles Move" in support of those affected by neuromuscular disorders. Muscular Dystrophy Canada is partnering with Safeway stores in Northwestern Ontario and Western Canada from July 31 - August 15, 2014, to raise funds to support the Safeway Mobility Grant program, post-secondary scholarships for young adults and to fund important research. Rachel, a 20 year old who lives in the surrounding area and is affected by muscular dystrophy says, "Wow thank you Safeway Canada, and your mobility grant funding. Because of you I was assisted in purchasing a piece of equipment to assist in moving my legs. Since I received the equipment I have become much more independent. It has helped me maintain my leg strength and has allowed me to strengthen both my body and mind. This exercise device has also greatly reduced my leg pain caused by cramping. It allows me to exercise as I need in my home, so I now can reduce the amount of time spent in the community going through physiotherapy. This equipment is not available in the treatment centers, hospitals or community physiotherapy clinics, … Continue reading →

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Thunder Bay Safeway Partners With Muscular Dystrophy Canada to "Make Muscles Move"

Posted: Published on August 2nd, 2014

THUNDER BAY, ONTARIO--(Marketwired - Aug. 1, 2014) - Muscular Dystrophy Canada and Safeway have launched their campaign to "Make Muscles Move" in support of those affected by neuromuscular disorders. Muscular Dystrophy Canada is partnering with Safeway stores in Northwestern Ontario and Western Canada from July 31 - August 15, 2014, to raise funds to support the Safeway Mobility Grant program, post-secondary scholarships for young adults and to fund important research. Rachel, a 20 year old who lives in the surrounding area and is affected by muscular dystrophy says, "Wow thank you Safeway Canada, and your mobility grant funding. Because of you I was assisted in purchasing a piece of equipment to assist in moving my legs. Since I received the equipment I have become much more independent. It has helped me maintain my leg strength and has allowed me to strengthen both my body and mind. This exercise device has also greatly reduced my leg pain caused by cramping. It allows me to exercise as I need in my home, so I now can reduce the amount of time spent in the community going through physiotherapy. This equipment is not available in the treatment centers, hospitals or community physiotherapy clinics, … Continue reading →

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Kenora Safeway Partners with Muscular Dystrophy Canada to "Make Muscles Move"

Posted: Published on August 2nd, 2014

KENORA, ONTARIO--(Marketwired - Aug. 1, 2014) - Muscular Dystrophy Canada and Safeway have launched their campaign to "Make Muscles Move" in support of those affected by neuromuscular disorders. Muscular Dystrophy Canada is partnering with Safeway stores in Northwestern Ontario and Western Canada from July 31 - August 15, 2014, to raise funds to support the Safeway Mobility Grant program, post-secondary scholarships for young adults and to fund important research. Rachel, a 20 year old who lives in the surrounding area and is affected by muscular dystrophy says, "Wow thank you Safeway Canada, and your mobility grant funding. Because of you I was assisted in purchasing a piece of equipment to assist in moving my legs. Since I received the equipment I have become much more independent. It has helped me maintain my leg strength and has allowed me to strengthen both my body and mind. This exercise device has also greatly reduced my leg pain caused by cramping. It allows me to exercise as I need in my home, so I now can reduce the amount of time spent in the community going through physiotherapy. This equipment is not available in the treatment centers, hospitals or community physiotherapy clinics, so … Continue reading →

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Dryden Safeway Partners with Muscular Dystrophy Canada to "Make Muscles Move"

Posted: Published on August 2nd, 2014

DRYDEN, ONTARIO--(Marketwired - Aug. 1, 2014) - Muscular Dystrophy Canada and Safeway have launched their campaign to "Make Muscles Move" in support of those affected by neuromuscular disorders. Muscular Dystrophy Canada is partnering with Safeway stores in Northwestern Ontario and Western Canada from July 31 - August 15, 2014, to raise funds to support the Safeway Mobility Grant program, post-secondary scholarships for young adults and to fund important research. On Saturday August 10th, between 11:30am - 1:30pm, Rachel who is 20 years old and lives in Dryden and is affected by muscular dystrophy will be at the local Dryden store, "Helping to Make Muscles Move" with dedicated Safeway staff and their customers. Rachel says "Wow thank you Safeway Canada, and your mobility grant funding. Because of you I was assisted in purchasing a piece of equipment to assist in moving my legs. Since I received the equipment I have become much more independent. It has helped me maintain my leg strength and has allowed me to strengthen both my body and mind. This exercise device has also greatly reduced my leg pain caused by cramping. It allows me to exercise as I need in my home, so I now can … Continue reading →

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CEO Reasserts Control Over Biotech Developer of Muscular Dystrophy Drug

Posted: Published on August 2nd, 2014

A few days ago, I posted an article about a biotech company calledSarepta Therapeutics (SRPT) that was experiencing potentially crippling internal acrimony. Things seem to be settling down, allowing Sarepta to get back to work seeking regulatory approval for a highly anticipated medication. Heres the background, framed in human terms, from my earlier piece: Imagine for a harrowing moment that your child has a debilitating, deadly disease for which theres no cure. A biotech company has a drug that might arrest your childs decline, maybe keep him out of a wheelchair, possibly add years or decades to his life. It would be toughno, unbearableto watch infighting at the company impede access to the medication. Its heartbreaking, Mindy Leffler told me by phone. Her son Aidan, 11, has Duchenne muscular dystrophy (DMD), an inherited muscle-wasting disease that afflicts 1 in 3,500 boys born in the U.S. DMD robs its victims of the ability to walk in early adolescence. Pulmonary or other complications typically kill sufferers in their twenties.Leffler was reacting to a regulatory filing and related news reports about the firing of a key executive atSarepta [which is] is seeking Food and Drug Administration approval for a DMD treatment that could … Continue reading →

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Beyond the tremors: Understanding the impact of Parkinsons disease

Posted: Published on August 2nd, 2014

By Shane Huntington Neuroscientist and neurologist Prof Malcolm Horne discusses Parkinsons disease, and examines new technological developments and the prospects they offer for early diagnosis and treatment of the condition. SHANE HUNTINGTON Im Dr Shane Huntington. Thanks for joining us. Among the many degenerative neurological disorders that can afflict us, Parkinson's disease is one such condition that can radically change the way we live and function. Second only to Alzheimer's disease in prevalence in the ageing West. Many of us have an image of the Parkinson's sufferer with increasingly debilitating tremors. But the symptoms of the disease go well beyond the visible. We don't know the cause, we don't have a cure, yet progress is being made. Today on Up Close we discuss research into Parkinsons and what the future holds for sufferers and those who support them. With new genetic technologies improving by the day are we are on the verge of cracking this disease? Can we identify the risk factors and try to avoid them? To answer these questions and more we are joined by Professor Malcolm Horne, Head of the Neurodegeneration Lab at the Florey Institute for Neuroscience and Mental Health, Consultant Neurologist at St Vincent Hospital … Continue reading →

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