Just Searching for Answers When diagnosed with a rare disease, cancer, or other illness, it makes sense that you’d research every type of treatment on the market. However, there are…
According to a story from globenewswire.com, the pharmaceutical company Portola Pharmaceuticals recently announced that its investigational product cerdulatinib has earned Orphan Drug designation from the US Food and Drug Administration…
According to a report by medicalexpress.com, researchers recently discovered a new genetic condition. Though researchers previously described the condition in animals, no human evidence had yet been found. Michigan State…
According to a story from Business Wire, the biotechnology company Abivax recently released the results from its Phase 2a clinical trial. This trial was testing its investigational product ABX464 as…
Misha Walker and her husband Mike have spent the summer in England, meeting with families who have children with a rare condition called arthrogryposis multiplex congenita (AMC). In the…
According to a story from Research & Development, increasing collaboration between a variety of different organizations is helping to propel and improve the develop of new drugs for the treatment…
According to a story from Mirror, a group of seven women who met each other through online support groups recently met in person for the first time. The women in…
According to a story from Disability Scoop, many military families that have children with debilitating rare conditions or special needs are having trouble getting the care that they need. Many…
Isaiah Austin is a name many sports fans may be unfamiliar with. In 2014 that may have been a different story. Austin played basketball at Baylor. Standing 7’1”, with surprising…
According to a story from pm360online.com, the global biotechnology company Shire plc recently announced its product Takhzyro (injectable lanadelumab) was approved by Health Canada after priority review. Takhzyro is indicated…
According to a story from ncb29.com, Tina Moore initially attributed her easy bruising to an infection. However, when she decided to get some blood work done, her doctors told her…
Happy Friday! As the week comes to a close, we want to spotlight four articles. We have advice from a patient with CVID who learned how to manage her condition…
According to a story from The New York Times, it didn't take long after Bertrand Might was born for his parents to realize that something was off. He had developmental…
A bill (H.R. 6801) has been introduced that is designed to support the development of rare disease therapeutics. Known as the Rare Disease Fund Act, it was introduced by Congressmen…
According to a story from news-medical.net, the new Evergreen Life health and medicines app could be a major benefit for patients across the UK, particularly patients with rare, chronic diseases…
According to a story from the Irish Times, Orla Tinsley was on the precipice of death before she received her lung transplant to treat her cystic fibrosis. She had to…
According to a story from blogs.bmj.com, the situation has been tense recently in the halls of the Cochrane Collaboration. Cochrane is a non-profit organization that involves the work of over…
According to a story from pm360online.com, the drug development company Cytori Therapeutics recently announced that its investigational product ATI-1123 has been granted Orphan Drug designation from the US Food and…
According to a story from BBC, there are only around ten people in the world who are currently living with a replacement bladder that was derived from their own cells.…
According to a story from The New Paper, the KK Women's and Children's Hospital (KKH) in Singapore has successfully developed a test that could make diagnosing genetic rare diseases more…
Three large black and white beasts rest under a shade tree to avoid the sun’s hot rays. They appear to be chewing gum, although they are really chewing their cud.…
According to a story from BBC, John Mason, age 80, is one of over 2,000 people in Wales who currently have idiopathic pulmonary fibrosis (IPF), a rare and ultimately lethal…
According to a story from Hematology Times, a recent study found that genetic sequencing after the completion of a hematopoietic stem cell transplant could provide doctors with vital information about…
The process of getting diagnosed with a rare disease, as well as living with one, can be a traumatic experience for patients. Many rare disorders or conditions may commonly cause…
According to a story from pm360online.com, the biopharmaceutical company Insmed Incorporated recently announced that the data from its Phase 3 clinical trial of amikacin liposome inhalation suspension (ALIS). ALIS was…
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