New Research to be Presented In just a few days the American College of Cardiology will host their 68th Annual Scientific Session (ACC.19). It will be held in New Orleans,…
Ontario, Canada is in the process of working to revamp their healthcare system. One of their primary goals is to increase the role that patients and their families/caregivers play in…
As much as we try to minimize human error in the healthcare field, it still happens. To decrease the chance of its occurrence we’ve created various policies, rules, and guidelines…
Inflammatory Bowel Disease Inflammatory bowel disease (IBD) is the collective term used to describe ulcerative colitis and Crohn's disease. These conditions cause diarrhea, rectal bleeding, fatigue, weight loss, and rectal bleeding. These…
Thanks to a grant from Worldwide Cancer Research, as well as support from various other organizations, researchers at UCLA recently uncovered a new, faster, easier, and more personalized way to…
Problems in China Around the world there are 7,000 rare diseases currently identified. Of these, researchers have uncovered effective treatment options for less than 10%. Sadly, even when diseases have…
The State of Rare Disease in India There are 7,000 known rare diseases worldwide. The Organization for Rare Diseases India (ORDI) estimates 70 million people are affected by a rare diagnosis…
SOJIA SOJIA stands for systemic-onset Juvenile Idiopathic Arthritis (JIV). It is a very severe form of JIA which causes joint pain, inflammation, recurrent fevers, and a skin rash. Patients often take predinsolone,…
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…
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…
Familial Chylomicronemia Syndrome Familial chylomicronemia syndrome (FCS) is a condition that is considered ultra-rare. It's caused when the enzyme called lipoprotein lipase (LPL) is impaired. It is can result in…
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.…
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…
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…
The very first patient in Medigene AG's clinical trial examining MDG1011 has officially received the treatment. This is the company's first human clinical trial for this TCR therapy. The trial…
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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