According to a story from the Tennessean, learning that both her husband and daughter had the rare and dangerous disease atypical hemolytic uremic syndrome (aHUS) was devastating for singer-songwriter Cameron…
According to a story from globenewswire.com, the drug development company Galectin Therapeutics, Inc., recently announced its plans to go forward with Phase 3 trials for GR-MD-02. The company is dedicated…
According to a story from BBC, Alex Hill was just eight-years-old when his mother, Sarah, started forming a hunch that something was off. The first warning sign came when Alex…
According to a story from EPM Magazine, the pharmaceutical company Orchard Therapeutics received Rare Pediatric Disease Designation for its gene therapy drug candidate OTL-200. The therapy is in development for…
According to a story from Financial Buzz, the pharmaceutical company Albireo Pharma recently announced that the first patient has been enrolled in its Phase 3 trial testing its experimental candidate…
According to a story from MD Magazine, the FDA has recently approved the first multiple sclerosis treatment catered to children with the disease. In this instance, the agency did not…
According to a story from news-medical.net, researchers working with the Institute for Experimental Pediatric Endocrinology of the Charité - Universitätsmedizin Berlin were able to successfully treat young patients that were obese…
According to a story from floridahospital.com, Huey Lewis, the front man and vocalist of the highly successful pop rock band Huey Lewis and the News, was recently diagnosed with Meniere's…
According to a story from azcentral.com, the Trump administration has decided to roll back some budget cuts that put the continued existence of regional Hansen's disease clinics in jeopardy around…
According to a story from The Independent, the 60 years of blood donations from a single man may be directly responsible for saving the lives of 2.4 million babies. The…
According to a story from cafepharma.com, Akcea Therapeutics, Inc. recently announced that the FDA's Division of Metabolism and Endocrinology Products Advisory Committee voted to recommend approval of the therapy Waylivra…
According to a story from medicalxpress.com, a recent study from the University of Birmingham discovered that the progression of multiple sclerosis could be predicted by spinal fluid analysis. This could…
According to a story from the Hereford Times, a combination of misdiagnosis and a critical mistake to forgo a simple blood test led to the tragic death of Callum Cartlidge.…
According to a story from Neuroscience News, the case of a woman with neck dystonia who was using a deep brain stimulation device called an implantable pulse generator (IPG) indicates…
According to a story from eurekalert.org, a recently study has helped reveal some of the genes that are associated with hemiplegic migraine headaches. About 15-20 percent of adults in the…
According to a story from eurekalert.org, the first patient that received treatment with alipogene tiparovovec reported positive results after an eighteen month treatment period. Alipogene tiparovovec is a gene therapy…
According to a story from the Leamington Observer, four-year-old George Young's first words were 'mama' and 'papa,' but he did not know how to say them for very long. George's…
According to a story from the Westside Gazette, it is essential for young men to be aware of the risk of testicular cancer and to know when it might be…
According to a story from The Tennessean, mother Melissa Hogan has been inspired by her son Case's Hunter syndrome to start a nonprofit organization called Project Alive. The primary purpose…
According to a story from Sickle Cell Anemia News, the St. Jude Children's Research Hospital just received $1 million in grant funding from The Links Foundation, which is one of…
According to a story from Newshub, Freda Evans learned early on that there were no treatments covered under the New Zealand health care system to treat her Pompe disease. Freda…
According to a story from blackpoolgazette.co.uk, the NHS has decided to postpone a decision to approve a drug for the treatment of Batten disease. This illness, which is fatal is…
According to a story from the Yorkshire Evening Post, five-year-old Toby Nye, from Leeds, has been seeing significant benefit from an antibody therapy treatment for his neuroblastoma. The therapy is…
According to a story from eurekalert.org, a recent test study suggests that patients with hemophilia B could be treated with just a single injection over the course of their lives.…
According to a story from Newswise, almost 500 infants that were born with congenital hyperinsulinism were treated with surgery that involved the removal of either part of or the entirety…
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