September is Krabbe Disease Awareness Month! Because a lot of times, these rare diseases are rarely talked about, it'll be useful to deep dive into the disease to learn more…
Amicus Therapeutics has met with the United States Food and Drug Administration to discuss the investigational drug AT-GAA, which is being researched as a potential treatment for Pompe disease.…
The political party the Scottish Liberal Democrats has voted in support of a motion about myalgic encephalomyelitis (ME). This motion calls for several changes, including stopping graded exercise therapy…
The United States Food and Drug Administration has awarded Fast Track Designation to an experimental gene therapy for the treatment of wet age-related macular degeneration. For more detailed information, you…
An international study led by researchers at the University of Alabama Birmingham has investigated the link between a specific genetic mutation underlying neurofibromatosis type 1, and the symptoms patients with…
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 report by the Irish Examiner, one in three people spend at least 45 hours or more caring for a someone living with cancer. Despite this being a…
September is Ovarian Cancer Awareness Month! How much about it do you know? Let's take a deep-dive. Ovarian cancer is a type of cancer that forms in a woman’s ovary.…
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 report by sciencealert.com, a novel form of antidepressant recently entered review by the FDA. While the new treatment is currently being examined for its effects on postpartum…
Corbus Pharmaceuticals have announced that they will go ahead with a Phase 3 study investigating the drug lenabasum as a potential treatment for dermatomyositis. For more information, you can view…
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…
A recently published study has highlighted the role of a sub-group of astrocytes (a type of brain cell) in brain inflammation, which can be a feature of multiple serious diseases,…
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…
With technologies like CRISPR, it is possible to imagine a world where genetic diseases fade into obscurity. Gene editing technologies may not have reached their potential yet, but many people…
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…
A report recently published by the American Association for Cancer Research has highlighted the progress made in fighting cancer but emphasises that continued investment by the government in healthcare research…
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…
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