According to an article from kait8.com, the pharmaceutical company Polaryx announced that its experimental drug PLX-200 has received Orphan Drug Designation from the European Medicines Agency (EMA) for the treatment…
According to a story from MSN, Heather Huus is living life without a stomach. This may seem impossible, but keeping her stomach could have made things far worse for her.…
According to an article from Khon2, locals on Honolulu Island in Hawaii are starting to raise concerns after the children of three firefighters from the Waipahu Fire Station were diagnosed…
According to a story from ABC News, medical researchers are beginning to monitor the long term health of people that have donated one of their kidneys. A kidney transplant can…
An exciting "win" in the relatively common glaucoma department could also mean a "win" for rare childhood glaucoma patients. According to an article from MedicalXpress, two recently approved treatments for…
According to a story from sixthtone.com, patients with hemophilia living in China are using paper cutting to help restore their dignity and reclaim control of their lives. Hemophilia is a…
According to a story from seas.yale.edu, a recent study conducted under the supervision of Yale University is providing new clues for the underlying cause of idiopathic pulmonary fibrosis. The researchwea…
According to a story from the National Organization for Rare Disorders, patient advocacy organizations are joining forces to send letters to Medicaid directors across the USA to advocate for the…
According to an article from Times-Call Regional News, a seven year old girl named Mila Makovec has just begun an experimental treatment for Batten disease. Originally from Boulder County, Colorado,…
According to a story from BBC, a former employer is paying for a woman's cancer treatment. 74 year old Pamela Stubberfield of Oxfordshire was diagnosed with mesothelioma. More than 80…
According to a story from Newswise, a recent study shows that Merkel cell carcinoma, which historically has affected only a few thousand people per year, is becoming more prevalent, especially…
According to a story from the International Business Times, researchers have successfully grown kidney tissue that can filter and produce urine within a living organism. This has the potential to…
According to an article from The Huddersfield Daily Examiner, Isla Sykes was diagnosed with mucopolysaccharidosis (MPS III), otherwise known as Sanfilippo syndrome, at age four. Her uncle Ryan Townrow, is…
According to a story from the Washington University School of Medicine in St Louis, scientists from the university used a sample of skin cells from diseased patients and transformed them…
According to a story from Yahoo News, The family of Isla Caton, a three year old girl diagnosed with neuroblastoma, has raised enough money for her to travel to New…
According to a story from tdn.com, Victoria Arlen recently discussed her past experiences with transverse myelitis and acute disseminated encephalomyelitis, which left her in a coma-like state for years. You…
According to a story from wncn.com, Seneca Ottey was diagnosed with esophageal cancer at sixty years old. Seneca was the father of Michelle Urban, who works as a pharmacist in…
According to a story from the Sun Herald, one in ten Mississippi residents suffer from one of the 7,000 diseases that are considered 'rare' in the United States. In the…
According to a story from brightsurf.com, a student at Clemson University discovered twenty-two different genes that are associated with the formation of glioblastoma, a rare and aggressive type of brain…
According to a story from independent.ie, acclaimed author Emma Hannigan announced that her final options for treatment had been exhausted in her fight with BRCA-mutated breast cancer. Her doctors have…
According to a story from CNNMoney, Scott Gottlieb, the current commissioner of the US Food and Drug Administration (FDA), said that he plans to curtail high drug prices and go…
According to stories from Wave3 News and WHAS 1, high schooler Jesse Schott of Bullitt County is in his third battle with brain cancer. At his local high school, Schott is…
According to a story from MedicalXpress.com, a team of Chinese researchers successfully regenerated lung tissue in a patient using stem cells transplants. Although it is still in its infancy, growing…
According to a story from BBC News, a total of around 20,000 people in the UK were born with spina bifida and major brain damage after their mothers were given…
According to a story from Business Wire, selumetinib, a drug in development for the treatment for neurofibromatosis type 1, was recently granted Orphan Drug Designation by the Food and Drug…
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