When you suffer with severe atopic dermatitis you can get pretty desperate to do anything to relieve the itch and other symptoms. So taking a bleach-bath is not out of…
Leukemia is a deadly serial killer that will claim more than 24,500 lives this year, but the Independent Citizens Oversight Committee of the California Institute for Regenerative Medicine (CIRM) just…
A new gene therapy treatment may be on the horizon for Oculopharyngeal Muscular Dystrophy patients. Find out more below, or keep up with the original story at musculardystrophynews.com. Oculopharyngeal Muscular…
A much-lauded piece of wisdom in engineering is that “failures teach more than successes.” While this might seem to contradict itself, I ask you to think back to your own…
For people suffering from severe atopic dermatitis (AD), commonly referred to as severe eczema, relief may be in sight, according to a recent article in Dermatology Times. If you're one of…
University of California, San Francisco scientists have developed a new vaccine that treats diffuse intrinsic pontine glioma (DIPG), a rare childhood brain tumor. To learn more about DIPG, click here. Great…
Each year pharmaceutical companies spend millions developing and testing potential treatments. These drugs have to go through many levels of testing to make sure they are safe and effective. And…
Whenever I was looking for something I had lost as a kid, my mom would offer the same, unhelpful advice: It'll show up in the last place you look for…
Great news for the rare disease community! Ultragenyx Pharmaceuticals reported back positive results from its phase 3 study of the treatment drug burosumab in adults with X-linked hypophosphatemia (XLH). XLH…
Our gut bacteria may be responsible for antiphospholipid syndrome (APS). What do you need to know about the ordinary gut bacteria known as commensals, and APS? Keep reading, or view…
Rett syndrome is a severe, genetic neurological disorder, which primarily affects girls. While it was once thought to be a form of autism, it's now understood as its own condition.…
African Americans face a far higher danger of developing multiple myeloma-- especially African American men, who run three times the risk of a diagnosis. These groups are also about half…
A mutant zebrafish that came into scientist's cross hairs over 20 years ago, has similar symptoms as patients who suffer from nemaline myopathy. This highly rare genetic muscle disorder is…
Sometimes, the only thing a rare disease needs is for technology to catch up with the times and save the day. Recently, this came in the form of three dimensional…
Sangamo Therapeutics shared some big news about the search for Mucopolysaccharidosis II (MPS II) treatment. They reported that they have, for the very first time, used experimental in vivo gene therapy to treat…
Good news for patients with Mucopolysaccharidosis Type VII (MPS VII)! In mid-November, the FDA approved a new drug for treatment of this rare disorder. The drug can be used for…
A major first step for a viable treatment option! Achillion Pharmaceuticals last week reported preliminary proof-of-concept results from its ongoing Phase 2, study of a new treatment for C3 glomerulopathy…
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, affects approximately 30,000 people in the United States. The National Organization for Rare Diseases estimates that 5,000 more people are diagnosed…
Everybody responds to treatments differently. The drug, diet, or surgery that successfully helps one person, may have a neutral or even negative effect on another. This is why the medical…
This past July, professionals, patients, and families with loved ones who struggle with primary hyperoxaluria Type 1, 2 and 3 were offered a glimpse of hope and a better future.…
Trigeminal neuralgia (TN) is the type of disease you wouldn't wish on anyone. It's characterized by extreme pain in the trigeminal nerve, which innervates the face. It's often described as…
Progeria is an incredibly rare genetic condition, where a random mutation causes a person to age rapidly. Progeria patients are essentially young people in the body of someone much older.…
Whenever I'm under a lot of pressure, I daydream about a day off, where I could watch true crime shows, eat pizza in bed, and never get out of my…
Since the birth of industrialization, societies have wrestled with the effects of pollution. Whether it contaminates the air, water, or soil, it can negatively impact human health, and scientists are…
Do you love cheese? Have an irrational fear of cats? Are able to wiggle your nose? As humans, we share more than a few surface-level characteristics with the common house…
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