Once upon a time there were three young brothers, and SPOILER ALERT- this part of the story, unfortunately, does not have a happy ending. Each brother suffered: Each had chronic…
Hey, do you guys remember that Mark Wahlberg movie that came out not long ago? You know, the one where he’s a cop? No no, not that one. The serious…
The term “superfood” is thrown around a lot. It is used for everything from salmon to spinach, from pistachios to pumpkin, from chia seeds to quinoa. When people use the…
"You're so full of sh*t." We've all heard it or said it at some point, be honest! But for one 22 year-old man in China, he was literally full of…
In April of this year, Rigel Pharmaceuticals, Inc. announced that it has submitted a New Drug Application (NDA) to the U.S. FDA for fostamatinib, a drug for patients with chronic…
With all the new products, widgets, and gadgets out there meant to clutter our life, there is one that manages to maintain style, grace, and cool points while providing a…
Attention hereditary angioedema patients: preventative treatment is on the horizon! Shire plc is one of the global leaders in rare disease research and pharmacology, and the group recently released positive…
My freshman year of high school, I began to get strange, red spots on my body. They initially developed on my arms, but within a couple of days, they had…
Want some more news, events and announcements on Batten disease? We got 'em! [one_half] [/one_half] [one_half_last] Pioneering Brain Surgery May Give This Toddler More Time FDA Press Release…
GW Pharmaceuticals is finding out... Epidiolex® (manufactured and tested by GW Pharmaceuticals) is a leading medical cannabinoid candidate for the treatment of rare, resistant, and early-onset epilepsy syndromes such as…
Here's the rundown on sickle cell anemia. It causes a person to have abnormal hemoglobin (sickle hemoglobin) in their red blood cells. In order to understand what I'm talking about,…
Peter Sadler has been raising funds to support the British Lung Foundation, which funds sarcoidosis research. The 42-year-old recently participated in the Bath-Half marathon. He's been on a mission to run in the…
In September, 2015, the U.S. FDA approved NUWIQ®, a recombinant antihemophilic factor (protein essential for normal blood clothing) developed by Octapharma AG, for the treatment of hemophilia A in children…
Okay. Hold the phone for just a minute. I’m a bit concerned after reading an article posted by a young girl who is living with sickle cell anemia. She basically…
“Whoever you are, I have always depended on the kindness of strangers.” Blanche DuBois may as well have delivered this as a tagline for every charitable organization in the world.…
Recently, when another rare mother and I were discussing the many trials that rare disease families face, we agreed that we wished that the general public would understand how vulnerable…
Want some more news, events and announcements on Lennox-Gastaut Syndrome (LGS)? We got 'em! [one_half] [/one_half] [one_half_last] New Research Could Revolutionize LGS and Other Disease Treatments LGS Patients…
I recently wrote about an 11-year-old Canadian girl who has an extremely rare disease called rapid-onset obesity with hypoventilation, hypothalmic dysfunction and autonomic dysregulation, or ROHHAD. There are roughly 100…
Big news for patients with BRCA-mutated metastatic breast cancer: a known ovarian cancer treatment has passed phase III clinical trials for BRCA-mutated metastatic breast cancer, and it's way better than…
Alexander Davis is from Tipton, Indiana. He has been living with spinal muscular atrophy (SMA). SMA prevents him from moving much and he is unable to walk. Therefore, he needs extra…
The concept of a “library” is something we probably take for granted—but at its core, the library is one of humanity’s greatest innovations. The act of collecting information for the…
Exciting news for people living with sarcoidosis! Vanderbilt University Medical Center (VUMC) has developed a Sarcoidosis Center of Excellence. Presently, Dr. Wonder Drake is the director of the center. She…
Elly Mayday, model, is teaming up with Adam Lawrence, filmmaker, to spread awareness and support for those affected by ovarian cancer. Elly was diagnosed with stage 3 ovarian cancer just…
Attitude is one of the most important elements of bringing together “who you are” with “what you do.” Attitude may not be everything, but it’s important enough that nothing else…
Type 0 is the worse type of spinal muscular atrophy (SMA). Fetuses that have this severe form of SMA typically die within the womb. A newborn can likely survive up…
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