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…
Olivia Egge is an osteosarcoma cancer survivor and an advocate for all those living with rare conditions like her own. The senior in high school living in Arlington, Virginia recently…
Spinal Muscular Atrophy Spinal muscular atrophy (SMA) is caused by a mutation in the SMN1 gene. This mutation results in a loss of motor neurons causing weak and atrophied muscles.…
According to a story from ALS News Today, ICD-10 disease codes will soon be up for revision in March 2019. What does this mean exactly? Well ICD stands for the…
How Liver Transplant Recipients are Selected Liver transplant recipients in the United States are selected according to their MELD score. This number is calculated from lab values. It aims to…
According to a publication from SMA News Today, Scotland is now the latest country to make spinal muscular atrophy (SMA) drug Spinraza available to patients at low or no cost…
Last year, Takeda, Eurordis, and Microsoft announced new collaborative efforts in the realm of rare disease. Specifically they were focused on the diagnostic period of rare conditions. However, up until…
Second Annual International Congress on Advanced Treatments in Rare Diseases The first International Congress on Advanced Treatments in Rare Diseases, otherwise known as RARE2018, was held in London, England. Now,…
What is Pulmonary Arterial Hypertension? Pulmonary arterial hypertension (PAH) is a rare form of high blood pressure. Normal blood pressure is around 14 mm HG at rest. Someone with PAH…
According to a publication from BioSpace, the French biopharmaceutical company Sanofi has successfully secured FDA approval for their acquired thrombotic thrombocytopenic purpura (aTTP) drug Cablivi. This represents the first approved…
Researchers at BGN Technologies (at Ben-Gurion University) have come up with a new way to predict and monitor progression of a wide array of neurodegenerative diseases. One of the illnesses…
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