According to a story from Science Trends, a recent trial of an experimental treatment demonstrates that it could be a potential treatment for Pompe disease. There are very limited treatment…
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…
According to a story from The Health Site, National Eye Donation Fortnight began in India on August 25th and will continue until September 8th. There are over a million people…
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…
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 Eye on the Cure, the biopharmaceutical company Ophthotech is hard at work researching and developing gene therapy solutions for a variety of rare retinal conditions.…
According to a story from Healio, a recent study found that irbesartan, marketed under the name Avapro, can help reduce the rate at which the aorta, the largest blood vessel…
According to a story from WXXI News, a startup from Rochester has set out to solve a dire medical problem that has global impacts on health. The problem at hand…
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…
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…
According to a story from Medical Xpress, three medical students at Florida International University are hoping to see if DNA collected from tampons could help detect ovarian cancer. There is…
According to a story from Bangor Daily News, Andrea Dole decided to have a home birth for James, her first child. With the support of her partner, Patrick, two midwives,…
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…
According to a story from Parkinson's Disease Today, a recent review highlights the potential benefits and limitations of telemedicine in treating Parkinson's disease. However, the authors also say that more…
The Amyloidosis Foundation is a non-profit organization that is committed to working and advocating for the amyloidosis patient community. The foundation is dedicated to supporting amyloidosis patients and their families…
According to a story from aawsat.com, a recent analysis suggests that nutrition therapy is an effective tool for slowing the progression of chronic kidney disease and delaying the need for…
According to a story from bioportfolio.com, the biopharmaceutical company Eureka Therapeutics recently released the results of the proof of concept trial for its experimental T-cell therapy ET140202 as a treatment…
According to a story from cnbc.com, a group of volunteers in a clinical study drank a concentrated brew that was filled with bacteria. This may sound unpleasant or even dangerous,…
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