When you or your child is first diagnosed with a rare disease it can be so difficult to know where to turn. You want to learn everything about the condition,…
Orencia Orencia (abatacept) is a treatment for juvenile idiopathic arthritis (JIA) that was approved for use in the U.S. for the pediatric patient population in 2008. In Japan, the therapy…
Short Bowel Syndrome Short bowel syndrome (SBS) is a rare condition which occurs when patients have a large portion of their intestine removed. The surgery which leads to this diagnosis…
The Word "Rare" Unfortunately, many experts in rare disease believe the phrase "rare disease" may be harming the very population it describes. The Director of the National Center for Advancing…
Coronado is a resort city located in San Diego, California. It's known for its luxury hotels, delicious restaurants, beautiful parks, and gorgeous beaches. It has just been announced that the…
SCID Severe combined immunodeficiency (SCID) is a rare disease which causes patients to have little to no immune response. With practically no immune system, SCID patients are very susceptible to…
The Origins of Fighting H.A.R.D. The Fighting H.A.R.D. Foundation was established in 2015 by two sisters who both live with multiple rare diagnoses. 16-year-old Allie is diagnosed with juvenile rheumatoid…
PREFER PREFER stands for Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle. It is a multi-stage project developed by Newcastle University's Welcome Centre for Mitochondrial Research in effort…
Phage Therapy Phages, or bacteriophages, are the natural enemy of viruses. They are the most abundant organisms in the biosphere. Researchers have used phages as treatments before, however after the…
The Growth of Precision Medicine Precision medicine is the idea that each patient is an individual, and therefore deserves an individualized treatment plan to secure the best outcomes. It is…
The FDA has just recently approved CYRAMZA as a treatment for those with hepatocellular carcinoma (HCC), who have been treated with sorafenib, and who have ≥400 ng/mL of alpha-fetoprotein (AFP).…
H.R. 2408 H.R. 2408 is a new piece of federal legislation otherwise known as the "Ensuring Access to Quality Complex Rehabilitation Technology Act." Essentially, this act creates a new category…
Scleroderma Scleroderma refers to a whole group of diseases, all rare, that cause the skin and connective tissues in the body to harden. This is a result of excess collagen…
Diffuse Intrinsic Pontine Glioma Diffuse intrinsic pontine glioma (DIPG) is a rare form of childhood brain cancer that has a 100% mortality rate. Children diagnosed with DIPG only typically live…
Researchers at the University of Colorado Anschutz Medical Campus have uncovered a never-before discovered immunodeficiency disease. How It Came About It started with a newborn boy who had a myriad…
Mantle cell lymphoma (MCL) and acute myeloid leukemia (AML) are two rare forms of blood cancer with a high unmet need. The majority of cases in these patient populations are…
Unknown Side Effects It's almost as if it's just a matter of time before a safety warning is issued for each new drug approved by the FDA stating an unexpected…
The first ever drug in the United States for treating transthyretin amyloid cardiomyopathy (ATTR-CM) has finally been approved by the FDA. It's amazing news for this patient community. But, you…
Cedric Rutland, a pulmonologist at Pacific Pulmonary Medical Group has shared his experience, research, and thoughts on cognitive bias in an effort to spread awareness for the issue, and hopefully,…
Transthyretin-Mediated Amyloidosis Cardiomyopathy The FDA has just approved the very first treatment for hereditary or wild-type transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM). ATTR-CM is a rare disease that occurs when amyloids, an…
The 2019 Congress of Clinical Rheumatology held May 2nd through the 5th in Destin, Florida featured a talk by Yusuf Yazici, the Director of the Behçet Syndrome Center at the…
Chronic Fatigue Syndrome Chronic fatigue syndrome (CFS) is a rare disease which causes, most notably, persistent fatigue that doesn't improve with rest. It is also sometimes referred to as myalgic…
Dutch Bros Coffee is hosting their 13th annual fundraiser for amyotrophic lateral sclerosis (ALS) this Friday, May 10th, 2019. It's called "Drink One for Dane" in honor of Dane Boersma,…
In February, Li Kegiang, Premier of the State Council, announced that the Chinese government was going to take steps to improve rare disease patients access to medicine. As part of…
What Exactly is Mast Cell Disease According to The Mastocytosis Society (TMS), there are three forms of mast cell disease. All involve the malfunction or over-development of mast cells in…
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