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…
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…
Most people diagnosed with ovarian cancer suffer from recurrences after treatment. And every one of them means less and less treatment options. It’s a disheartening, never-ending cycle. What has stumped…
Hannah Hawkins has an autoimmune disorder called transverse myelitis. She has to go to physical therapy multiple times each week to help her walk. Using a crutch is the norm…
At Patient Worthy, we’re all about rare diseases, and the focus of almost all our posts is the patient. Today, I want to shine a bright, white light on all…
In collaboration with the Hereditary Neuropathy Foundation (HNF), Patient Worthy asked members of the Charcot-Marie Tooth (CMT) community to tell us their stories, in hopes of inspiring everyone in the rare community…
Think all those fundraising walks you see advertised don't help? Think again! Shelly Meitzer, the mother of two children with tuberous sclerosis complex (TSC), a rare multi-system genetic disorder, has…
The First Ever Seattle Rare Disease Fair happened on June 3rd, 2017. And it was a BLOOMING success. The goal of the event was to spread awareness of rare disease…
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