Hannah Evans, a 27-year-old woman from South Wales with several rare diseases, has reached her £37,500 fundraising goal that will support her trip to America to access an experimental and…
Lisa Clayton has been diagnosed with cancer as many times as she's given birth. She's a mom of 5, and she has just heard the news "you have cancer" for…
September was Childhood Cancer Awareness Month and Sickle Cell Awareness Month. To mark to occasion, the Aflac Cancer and Blood Disorders Center at the Children’s Healthcare of Atlanta released a…
HPV or human papillomavirus is the most common sexually transmitted disease in the US. It is so common that most sexually active people will catch the virus at some…
According to a story from Boston Health News, The US Food and Drug Administration's Accelerated Approval program is being used to approve a steadily growing number of drugs in recent…
Researchers have previously found that patients with cancer who live in more rural locations have worse outcomes than those from urban places. However, it isn’t clear what causes this pattern.…
October is Trigeminal Neuralgia Awareness Month! One way you can honor this month is by signing TNnME: Intl Trigeminal Neuralgia and Me's petition for the World Health Organization (WHO) to add Trigeminal Neuralgia…
A new neuromuscular research center has opened at the Ottawa Hospital. The new center will allow patients living with neuromuscular conditions, such as muscular dystrophy and ALS, to be a…
According to a story from PR-Inside, the biotechnology company Saniona recently announced that the second part of its Phase 2a clinical trial testing Tesomet as a treatment for Prader-Willi syndrome…
The United States Food and Drug Administration has awarded Fast Track designation to an investigational drug called PTG-300 for treating chronic anaemia caused by ineffective erythropoiesis in patients who have…
Rare diseases come in many forms, shapes, and sizes - with varying degrees of symptoms and attributes. Here is one that made news on the Internet, for it's peculiar and…
According to a story from HD Buzz, a recent study has conducted an in-depth investigation into juvenile onset Huntington's disease. It is quite unusual for this juvenile onset form to…
According to a story from The Atlantic, the continual development of gene editing technologies like CRISPR have the potential to cure a substantial portion of rare diseases that are linked…
According to a story from Scoop Independent News, speech therapists from the Waikato District in New Zealand have been using telehealth technology to great effect in order to deliver speech…
A study carried out by researchers from the University of North Carolina at Chapel Hill suggests that doctors in the state may not be testing patients for Ehrlichia infection often enough.…
LiPlaCis is a formulation of cisplatin that delivers its agent directly to cancerous tumor sites. Its efficacy has so far only been studied within breast cancer patients. However, scientists have reason to…
Actress Marcia Cross, best known for her role as Bree Van de Kamp on Desperate Housewives, has shared that she is now healthy after battling anal cancer in a series…
According to a recent press release from Bluebird Bio, the European Medicines Agency has accepted and will review their marketing authorization application (MAA) for the investigational gene therapy LentiGlobin™ as…
According to a story from MarketWatch, the biopharmaceutical company MediciNova recently announced that the company's product ibudilast was granted Orphan Drug designation by the FDA. The designation was granted for…
If you think you have to be middle-aged or older to check your cholesterol, think again. Laura Livesey is a relatively young blogger (you can check out her blog, Confidence…
A drug called Coversin is being investigated as a potential treatment for four orphan diseases: bullous pemphigoid (BP), atypical keratoconjunctivitis (AKC), thrombotic microangiopathies, and paroxysmal nocturnal hemoglobinuria (PNH). Recently, new…
Happy October, everyone! We hope everyone's enjoying this new ~fall weather~ and the endless supply of pumpkin spice flavored food products. This week, we're highlighting four FDA announcements we think…
PharmAbcine has announced that the United States Food and Drug Administration has responded to the Investigational New Drug application they submitted on behalf of an experimental drug called TTAC-0001, which…
According to a story from 9news.com.au, the first sign that Jemima Leydon had ovarian cancer was a stomachache. Her mother Megan assumed that it was just a stomach bug that…
A new drug designed to treat MAC lung disease as part of a combination antibacterial drug regimen in adults with limited treatment options has been granted Accelerated Approval by the…
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