According to a story from globnewswire.com, the gene therapy company Adverum Biotechnologies recently announced that the US Food and Drug Administration (FDA) has granted them Orphan Drug designation for their…
According to a story from Medical Xpress, a team of scientists from Michigan State University have managed to reveal the manner in which a genetic malfunction can lead to the…
According to a story from BioSpace, the US Food and Drug Administration (FDA) recently approved the drug Oxervate (cenegermin). This treatment is the first FDA approved therapy for the rare…
According to a story from medicalxpress.com, a team of scientists at the University of Massachusetts Medical School have successfully developed a strategy for editing genes that could be used to…
According to a press release from Eisai Inc. and Merck (also known as MSD), Lenvima®(lenvatinib) has been approved by the United States FDA as a first-line treatment for patients…
According to a story from mirror.co.uk, Vanessa Riddle, who had the rare cancer neuroblastoma, recently died after defying her cancer for almost a decade. She passed on June 1st, with…
According to a story from the St. Helens Star, Amanda Sackville-Wiggins was diagnosed with hepatocellular carcinoma, a rare type of liver cancer, just after her 21st birthday. The cancer was…
According to a story from BioPortfolio, the U.S. Food and Drug Administration (FDA) has recently approved a new combination therapy for the treating small lymphocytic lymphoma and chronic lymphocytic leukemia…
According to a story from the San Francisco Chronicle, Isabel Bueso's parents were told when she was born that she would not live more than seven years. However, she continued…
Happy Back 2 School Time! As the week comes to a close, we want to spotlight four articles. We have an honest piece from PW contributors Denise Crompton and Tom…
According to a story from Tablet Magazine, Emily Kramer-Golinkoff is a 33 year old woman who is living with cystic fibrosis, a rare disorder that primarily affects the lungs. Emily…
According to a story from BioSpace, Martin Pharmaceuticals' Livantra appears to be on something of a roll. Earlier in the year, the drug received Orphan Drug designation for the treating…
No one likes to be judged. However, during the many years that I've been involved with rare diseases families, I've learned that some people are very quick to judge parents…
According to a story from Newswise, a team of scientists from the St. Jude's Children's Research Hospital have been hard a work conducting an in-depth analysis of a pediatric solid…
According to The Times of Israel, a group of researchers believe they have found the origin of what could lead to a treatment for amyotrophic lateral sclerosis. Given the debilitating effect…
The healthcare technology company RDMD was founded by Onno Faber, who has himself been diagnosed with a rare disorder. RDMD, which describes itself as “dedicated to accelerating research” with…
According to a story by The Japan Times, Masatane Muto relies on a power wheelchair to move around. He is hardly in control of his own body and requires help…
According to a story from Charcot-Marie-Tooth News, a recent study has managed to identify new clinical evaluations that will allow doctors to more effectively monitor the condition of patients with…
According to a story from OncLive, the results of a Phase II trial for an experimental drug called SL-401 showed that the product could be a potentially effective treatment for…
According to a story from Morningstar, the company Alexion Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) has agreed to review their Biologics Licensing Application for their…
According to a story from USA Today, despite numerous warning letters sent from the US Food and Drug Administration to the offices of doctors and other medical professionals across the…
The United States FDA has approved the first generic version of EpiPen and EpiPen Jr (epinephrine). The approval was granted to Teva Pharmaceuticals USA, who will now be able to…
According to a story from CheckOrphan, Durhane Wong-Rieger, the CEO of the Canadian Organization for Rare Disorders, says that the Canadian health system has been letting down the roughly three…
Elmo and friends are here to help! - thanks to the good people of The National Association of Community Health Centers (NACHC)! For those who have never heard of the NACHC,…
Gabriel Low, a 17-year-old from Hawaii, just completed a 3,000-mile bike ride across the country. He’s been riding since June 11th when he embarked from Seattle, WA to raise awareness,…
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