Consider for a moment that your life is dominated by spontaneous nosebleeds, chronic stomach hemorrhaging, persistent anemia and a complex constellation of other manifestations, some potentially life-threatening.
Recent advances in the development of cancer therapies have increased long-term survival and prognosis. However, the increased burden and prevalence of harmful side effects, including cardiomyopathy, have emerged alongside ...
Apr 10, 2019
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A Bangladeshi dubbed "Tree Man" because of bark-like growths on his skin will need aggressive surgery to remove them, doctors said Tuesday, after dozens of attempts failed to cure the extremely rare genetic condition.
Jan 22, 2019
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Fresh discoveries about a rare genetic disorder that affects mainly boys may inform the development of therapies to treat the condition.
Dec 12, 2018
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Clemson University faculty have been awarded a grant from the National Institutes of Health to investigate the use of 3-D imaging that could allow for quicker diagnosis of children with Phelan-McDermid syndrome, a rare genetic ...
Oct 10, 2018
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New research led by the Murdoch Children's Research Institute (MCRI), has combined gene editing technology with stem cell kidney regeneration to correct a patient's gene mutation.
May 4, 2018
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A year ago Abul Bajandar, a Bangladeshi rickshaw-puller dubbed "tree-man" for the bark-like growths that once covered his body, appeared to have made a full recovery from the rare condition that has afflicted him for a decade.
Feb 2, 2018
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Researchers at Sanford Burnham Prebys Medical Discovery Institute (SBP) have led a preclinical study demonstrating that the drug palovarotene suppresses the formation of bony tumors (osteochondromas) in models of multiple ...
Nov 20, 2017
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A clinician who treated Charlie Gard, the terminally-ill British baby who died last month, said Saturday his short life became a soap opera fuelled by President Donald Trump and the pope.
Aug 5, 2017
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Charlie Gard, the terminally-ill British baby whose plight drew sympathy from Pope Francis and US President Donald Trump and sparked a debate about medical ethics, died on Friday, his mother said.
Jul 28, 2017
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