According to a story from finanznachrichten.de, the Swedish drug company Calliditas Therapeutics has recently announced that the US Food and Drug Administration (FDA) has given one of the company's experimental drugs…
According to a story from Rare Disease Report, the US Food and Drug Administration (FDA) has announced its approval of a combination therapy from Janssen which consists of ibrutinib plus…
According to a story from nbcwashington.com, Nikki Ferraro was diagnosed with a rare type of thyroid cancer when she was just seventeen years old. While she was able to survive…
According to a story from BioSpace, the biotechnology company REGENXBIO, Inc. recently announced that its experimental gene therapy candidate called RGX-181 has been granted Rare Pediatric Disease designation from the…
According to a story from independent.co.uk, organ transplants have been responsible for saving many lives. Organ transplants are often a critical part of treatment for a lot of rare diseases.…
According to a story from Reuters, a new website developed by the American Gastroenterological Association is meant to help women that have inflammatory bowel disease have safe and healthy pregnancies.…
According to a story from WebMD, the rare nervous system disorder chronic inflammatory demyelinating polyneuropathy is notorious for being difficult to diagnose. Many rare diseases can be challenging to identify.…
According to a story from Medical News Today, leaving ulcerative colitis (a form of inflammatory bowel disease) untreated could result in dire consequences for a patient. While ideally all patients…
According to a story from mirror.co.uk, a five-month-old girl named Maryam Malji was diagnosed with spinal muscular atrophy, a progressive, degenerative disease that is ultimately fatal without treatment. Her brother…
According to a story from GoodRx, hyperkalemia, which is a condition of elevated levels of potassium in the blood, can be a potentially life threatening situation. The condition can appear…
According to a story from labiotech.eu, the biotechnology company Orphazyme has been developing an experimental therapy for Niemann-Pick disease type C. In a recent Phase 2/3 clinical trial, the investigational…
According to a story from arkansasonline.com, Rep. Julie Mayberry R-Hensley of the Arkansas General Assembly is sponsoring House Bill 1074. This legislation proposes adding the rare and potentially fatal genetic…
According to a story from the Tenterfield Star, a fundraiser was recently held for Tommy Johnston, a one year old boy who was diagnosed with the rare Niemann-Pick disease type…
According to a story from The New York Times, a new wave of research is starting to reveal the close connection between the human gut microbiome and the brain. Some…
According to a story from falmouthpacket.co.uk, couple Geoff and Judie Davis were devastated when their two year old boy Mikey died after being diagnosed with neuroblastoma. That was more than…
According to a story from BBC, it is becoming increasingly clear that a lot of the common phrasing and language that we use to talk about cancer and cancer patients…
According to a story from 2 Minute Medicine, a recent analysis found that patients with lupus nephritis saw their risk of death decrease by 70 percent on average after receiving…
A rarely addressed subject that comes up in discussions about people with disabilities is the subject of romantic relationships, intimacy, and parenting. More than likely, a lot of day to…
According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Processa Pharmaceuticals, Inc. recently announced that the first patient had been dosed in the company's Phase 2 clinical trial. This trial…
According to a story from globenewswire.com, the biopharmaceutical company Xenon Pharmaceuticals, Inc., recently announced the latest updates on the progress of two of its experimental products. The first is called…
According to a story from prweb.com, researchers from the Johns Hopkins Kimmel Cancer Center and the Allegheny Health Network (AHC) Cancer Institute have entered a partnership that plans to investigate…
According to a story from pm360online.com, the drug developer Innate Pharma SA recently announced that its experimental therapy IPH4102 has been granted Fast Track designation from the US Food and…
According to a story from wired.com, everything changed for Sonia Vallabh and her husband Eric Minikel after Sonia's mother died from a mysterious illness. Sonia's mother Kamni was in her…
According to a story from Science Daily, a team including Dr. Stephen Meltzer from the Johns Hopkins University School of Medicine has developed a method for detecting esophageal cancer. In…
According to a story from the Irish Mirror, 32-year-old Ben McGuire recently sued the HSE after claiming that the Cork University Hospital was unable to detect a pancreatic cancer tumor…
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