According to a story from Kamloops Coffee Talk, Darrin Rein, of Kamloops, British Columbia, was just recently diagnosed with spinocerebellar ataxia, a rare disease that affects mobility and coordination. Despite…
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…
May is Healthy Vision Month! Rare disease or not, maintaining healthy eye health should be a priority for all of us. The National Institute of Health has resources for not…
According to a story from BioSpace, the drug developer Biogen recently released interim results from a Phase 1/2 clinical trial. This clinical trial is testing the drug tofersen, which is…
Behcet’s Awareness Day is May 20th! Behcet’s disease (also known as Behçet’s and Behçet’s syndrome) is a rare condition that causes chronic inflammation which manifests as ulcers (commonly referred to…
I was diagnosed with benign essential blepharospasm at the end of 2015. This is a neurological condition which causes the muscles around my eyes to contract, resulting in uncontrollable blinking…
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…
According to a story from globenewswire.com, the biopharmaceutical company Poseida Therapeutics, Inc. recently announced that the company's investigational product candidate P-BCMA-101 has been granted Orphan Drug Designation from the US…
Happy Wednesday! We're officially halfway through May if you can believe it! This week we're highlighting a piece about an intern using their rare experience to inform their work, followed…
According to a story from apnews.com, the drug developer Ayala Pharmaceuticals has recently announced that the company's investigational product candidate, known as as AL101, has earned Orphan Drug Designation. This…
According to the Wall Street Journal, the National Institutes of Health (NIH) has reversed its initial decision to prevent two of its researchers from meeting with federal investigators to comment…
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…
David Rowland, age 77, is a cancer survivor and former veteran of the Air Force. He is also a tube feeder. People who use a feeding tube are considered disabled…
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…
According to a story from Specialty Pharmacy Times, data from a Phase II clinical trial has recently been released which suggests that the drug tagraxofsup is a useful treatment in…
According to a story from radio.wpsu.org, soprano singer Charity Sunshine Tillemann-Dick recently passed away at the young age of 35 because of pulmonary arterial hypertension, a rare condition of increased…
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…
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…
As I listened to her story I kept thinking, “You can’t make this stuff up.” Or at least no one would want to anyway. The first time I met Adeola…
According to a story from EurekAlert!, a team of scientists affiliated with the University of Cambridge have pioneered a new test that could herald a new development in the treatment…
According to a story from Chemical & Engineering News, the American Society of Gene & Cell Therapy annual meeting was more crowded than usual this year. In fact every event…
Happy Thursday! We hope everyone is enjoying this beautiful spring week! This week we're highlighting a story about a partner of ours that helps support creativity in kids with rare…
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