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…
There is a must-read for more inspiration about living with sickle cell anemia (SCA) – a young woman’s blog, but not just any blog. She’s a DIVA (as in, absolutely…
BrainStorm Cell Therapeutics Inc. is looking to begin producing NurOwn® adult stem cells for their phase III clinical study on ALS patients. The therapeutic company is hoping that the development of…
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.…
Though they are frequently misunderstood, genes play an important role in our lives. Most people know that they inherit the majority of their physical features from the genes their parents…
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…
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