On February 24, 2019, the Organization for Rare Diseases India, USA (ORDIUSA) held a 7km walk/run at four different locations across the United States. The Event was called Racefor7 and its goal…
Each year, Rare Disease Day (February 28th) incites individuals and organizations to investigate and reform their rare disease efforts. This year, the EU Commission has made drastic changes which should…
According to a story from Ottawa Citizen, the CHEO Research Institute has recently begun operations at its Centre for Genomic Innovation. Much of the the center's operations will focus on…
On December 28, 2018, five-year-old Oscar Saxelby-Lee was diagnosed with a rare and aggressive form of leukemia. Now he's in a race against time to find an eligible stem cell…
Happy Thursday! Today, we're highlighting an article on a story about rare disease discrimination, followed by news about a musician who went public with a rare diagnosis. After that, we…
A Collaborative Effort An international team of researchers has just uncovered the genetic cause for an ultra-rare form of leukoencephalopathy. Leukoencephalopathy refers to a family of conditions which affect the…
About GOSH The Great Ormond Street Hospital (GOSH) is both a pediatric hospital, treating many children with rare conditions, and a charity organization for patients like those they care for.…
The European Medicines Agency (EMA) recently published a press release in which the agency's human medicines committee, known as CHMP, as recommended the authorization of a new treatment for a…
According to a story from Central Charts, the biopharmaceutical company Acceleron Pharma Inc. recently announced that the company's experimental drug candidate ACE-083 has earned Orphan Drug designation from the US…
According to a story from The Muscular Dystrophy Association (MDA), the organization alongside the Charot-Marie-Tooth Association (CMTA) has awarded grant funding totaling $276,430 to a research study that will develop…
According to a story from Markets Insider, Taiho Oncology, Inc. recently announced that the US Food and Drug Administration (FDA) has officially approved the company's cancer drug LONSURF in the…
According to a story from news-medical.net, the University Hospitals in Cleveland, Ohio and the University of Oxford have recently announced their plans for a multi-year affiliation. The goal of this…
Not only are rare diseases well, rare, but the same rare disease can also affect different patients in a multitude of ways. For instance, some diseases can impact many different…
Jordan Flake and her son Jackson were on their way from El Paso, Texas to Dallas, Texas when they were kicked off their flight because of their Ichthyosis diagnosis. They were on…
Ipsen has just announced that they will be acquiring Clementia Pharmaceuticals. This merging of companies is exciting for the rare community because it will strengthen the resources, knowledge-base, and overall…
According to a story from Hemophilia News Today, the drug developer Spark Therapeutics, Inc., has recently released several updates in regards to a number of its experimental gene therapies that…
ADA-SCID ADA-SCID is a form of Severe Combined Immunodeficiency (SCID) which is caused by Adenosine Deaminase Deficiency. A lack of the ADA gene in the body results in impaired development of the…
According to a story from BioSpace, the biopharmaceutical company DelMar Pharmaceuticals, Inc., has recently announced an update to its Phase 2 clinical trial. This clinical trial is testing the experimental…
Happy Rare Disease Week! This week has been all about rare diseases. Today, we're highlighting an article from a PW contributor with FAP, followed by a story describing some shortcomings…
Lupus Nephritis Lupus nephritis (LN) is one of the most common complications of systemic lupus erythematosus (SLE). It affects approximately 100,000 patients in the United States alone. Basically, the patient's…
Proteus Syndrome Proteus syndrome is a rare disease caused by the overgrowth of tissues including the skin, adipose tissue, and parts of the central nervous system. It also affects the…
This is the story of five-year-old Aubree Fox who has recently been diagnosed with hydrocephalus, stenosis, and an aneurysm in her brain. This slew of life-threatening diagnoses were all a mystery…
NICE stands for the National Institute for Health and Care Excellence in England. This organization is responsible for evaluating the cost effectiveness of all medications as they come to the…
For years the accepted estimate of multiple sclerosis (MS) patients living in the United States was 400,000. A recent study, funded by the National Multiple Sclerosis Society (NMSS) has confirmed that number is…
Diamond Blackfan Anemia Diamond Blackfan anemia (DBA) is a rare form of anemia caused by a genetic mutation in the bone marrow. This mutation results in a reduced production of…
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