Spark Therapeutics will be hosting a conference call this Tuesday. For those interested in the business side of therapy, this is an interesting opportunity to observe. Find out more about…
According to an announcement from ema.europa.eu, the European Medicines Agency (EMA) recently granted market authorization for a new treatment for alpha-mannosidosis. The new drug is an enzyme replacement therapy called…
Maddie Holt from Everett Washington is a 5-year-old with severe disabilities. She can barely hear or see, and she certainly can't walk or talk, reports Maine Public. Unfortunately for her,…
Andy Trapp is a videographer who runs AB Trapp Productions, a small business documenting weddings and family occasions. He has been living with uncontrolled PKU since the age of 8.…
According to an article from BBC News, Jackie Harrison had a daunting choice to make. There was a fifty percent chance that she would contract Huntington's disease, a rare condition…
According to USA Today, a new study suggests that pregnant women should be conscious of their diet and avoid any restricting diets that include low-carb and gluten-free limitations. The study…
According to a story from the National Hemophilia Foundation, the pharmaceutical company Novo Nordisk announced that its product REBINYN was now available for hemophilia B patients. The treatment is intended…
According to a story from CBS News, Hazel Hammersley was diagnosed with neuroblastoma at age two. As a result, she was forced to fight for her life at a very…
According to a story from Bronchiectasis News Today, the results of a new study suggests that bronchiectasis may trigger autoimmune inflammatory disease. The study found that the condition was frequently…
A severely aggressive brain tumor in 19-year-old Callum Hardyman has forced his brain to turn sideways, reported The Sun. Callum suffers from a terminal brain cancer called glioblastoma. To learn…
The Orphan Disease Center in association with the University of Pennsylvania and the Loulou Foundation just announced their 2018 Pilot Grant Program for CDKL5 Deficiency Disorder. CDKL5 deficiency disorder (CDD)…
Just this last June, a new drug successfully used to treat metastatic breast cancer patients in England, has been denied for a third time by Scottish Medicines Consortium (SMC), as…
The Alsabaa family not only has to deal with their 6-year-old son ,Tony, fighting children's idiopathic arthritis, but they are also not allowed to be in the States with him…
According to a story from CTV Vancouver Island News, a charming young child known affectionately as 'bus boy' died in his sleep on Saturday, January 27th. Dominic Orr was only…
Shaquem Griffin, a linebacker from the University of Central Florida receives a ton of buzz because he's an amazing athlete. Point blank. As he should. Originally reported by Rare Disease…
In an article posted by the Wyoming Medical Center, Dr. Adam Linck clears up some common questions concerning hereditary hemochromatosis. People living with this condition have an excess amount of…
While she's constantly smiling and full of energy, little girl Makenzie Cadmus struggles with a rare condition known as epidermolysis bullosa, reports News 12 Long Island. The rare disease has…
For people who don't live with chronic painful conditions, such as hepatic porphyria, it's hard to imagine the type of intense pain Colin McEwen was in -- both physically and…
A wealthy do-gooder is championing Idiopathic Pulmonary Fibrosis (IPF). No, it's not Tony Stark (a.k.a. Ironman). But there is a mega-force super power uniting global innovators, researchers, and healthcare entrepreneurs…
Just recently reported by BBC News, Jake Coates made a hard decision to stop attempting to have a child through the surrogate route after two failed attempts. Not even two…
Sophia Campa-Peters, a little 9-year-old suffering from a rare disease that resulted in several strokes, had a successful brain surgery just a few weeks ago, as reported Lubbock Online. The…
University of Glasgow scientists might have just come across an incredible new approach to providing patients with pharmaceutical medications on demand, reported first by International Business Times. This new technology…
16-year-old Teenager Ciara Micks of Limerick, Ireland, tries to remain positive despite an unsettling situation regarding next steps to treat her EDS, Ehlers-Danlos Syndrome, reports Limerick Leader. Ciara deals with…
11-year-old Nolan Cyr is not like anyone else his age. When facing round after round of chemo in the hospital to fight his cancer, it wasn't himself he was thinking…
According to a story from Independent, cumbersome delays and possible funding cuts are severely hampering England's National Health Service (NHS). These delays are proving very costly for patients with long…
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