According to a story from Charcot-Marie-Tooth News, early preliminary testing of a possible gene therapy treatment for a certain type of Charcot-Marie-Tooth disease showed potential in a mouse model. This…
Personalized medicine is the latest craze in patient care. In essence, it refers to the development of treatment options which take into account the patient as an individual. It reduces…
According to a story from Pulmonary Hypertension News, the drug developer United Therapeutics recently issued a statement in which the company announced that it was ceasing the development of esuberaprost…
Happy Thursday! We hope everyone's month is going well! This week, we're highlighting two stories about drug pricing and reimbursement. Next, we have an article on mastocytosis diagnoses and (yet…
Upcoming Conference The International Society for Mannosidosis & Related Diseases (ISMRD) serves as an international patient advocate for those living with glycoprotein storage diseases. These include alpha-mannosidosis, fucosidosis, mucolipidosis type…
According to a story from AJC, three year old Reid Peteet is one of over 550 kids that have been diagnosed with acute flaccid myelitis since 2014. 2018 saw a…
Six weeks ago, Gretchen and Josh of Oklahoma rushed their precious 5-month-old Opal to the hospital after she became extremely sick from a cold. It didn't take doctors too long…
Danon Disease Danon disease (DD) is an extremely rare condition that is not very well understood in the medical community. It was first described in 1981, and it is named…
A three-year research grant of $11 million has been awarded through the Gilbert Family Foundation in America. According to a recent article in News-Medical Life Sciences, the Foundation was formed…
According to a press release from Inovio Pharmaceuticals, the American biotech company's experimental recurrent respiratory papillomatosis (RRP) drug INO-3106 showed promising results at the end of a pilot clinical study…
According to a story from Xconomy, a recent report from the Institute for Clinical and Economic Review (ICER), based on Boston, is calling for reductions to the price tags of…
According to a recent article in Check Orphan, genetic and biochemical testing are combined in a new method of metabolic testing called the CentoMetabolic panel. The announcement comes from…
Mastocytosis Mastocytosis is a rare disease caused by mutations in the KIT gene. The condition causes mast cells to accumulate in the body. Depending on the location of these immune…
According to a publication from health website Stat, a new way of thinking about the price of drugs is taking root in Washington D.C. and beyond. "Value-based agreements" are a…
According to a story from Cystic Fibrosis News Today, Elizabeth Rogers, a cystic fibrosis patient, is just one of the many who have been disappointed by the lack of impact…
According to a press release from American biotechnology company Soligenix, the European Medicines Agency's (EMA) Pediatric Committee recently agreed to the Company’s Pediatric Investigation Plan (PIP) for experimental oral mucositis…
According to a press release from the Muscular Dystrophy Association published at Biospace, the group has resolved to award eight new research grants for the study of amyotrophic lateral sclerosis…
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An article in BioSpace reports new information published online by the NEJM concerning Hirschsprung disease. The disease involves the absence of nerves in parts of the intestines prior to birth. Researchers…
My first admission is that I actually know quite a bit about ABC's The Bachelor. Yes, that includes The Bachelorette too. I first started watching “ironically“ with my girlfriend, who…
Nigam Shah and Purvesh Khatri, professors at Stanford University, along with graduate student Madeleine Scott have just announced results from a research study which could be pivotal for those living…
Happy Spring! Anyone else got that spring fever? Unless you're getting tortured by allergies, it is a great time to be outdoors. The bees are buzzing, the flowers are blooming,…
Tracheostomies in ALS A tracheostomy is essentially an invasive procedure which helps patients breathe. It also removes secretions from their lungs. Doctors cut an opening into the patient's neck and…
According to a story from Radio NZ, two grandmothers have taken a central role in getting a petition delivered to the parliament of New Zealand. This petition is calling for…
According to a story from CheckOrphan, there are currently very few effective treatment options for chordoma, a rare, slow growing tumor that originates from bone. However, a recent research discovery…
RADOIR Rare Diseases Foundation of Iran (RADOIR) is an organization dedicated to supporting those living with rare diseases in Iran. It is a nonprofit which was founded in 2008 with…
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