Below you'll find a list of all posts that have been tagged as “rare disease”
In 2006 when Orphan Drug Solutions first went to Viropharma’s offices (prior to them being acquired by Shire), they had 80 employees dedicated to the advancement of their primary drug, Cinryze. Cinryze was and is the only FDA-approved C1 esterase inhibitor therapy used to treat HAE (Hereditary Angioedema), so its success was not just important for them as a company, …
While there are many negative stories floating around about the youth of our country, it is always exciting to come across a story that literally puts a smile on your face. An 8-year-old boy has raised over $800,000 for his friend who suffers from a rare disease. That headline alone made my jaw drop. Lora Purtnazarian and her husband Rabin …
We are super excited for our friends at Global Genes as they launch RARE Daily! Global Genes’ RARE Blog has been re-imagined to bring more opportunities for rare disease patients to learn and stay informed on important issues with a team or new contributors and subject-matter experts within the rare disease community. “The new format of the RARE Daily provides …
I recently came across this article while browsing the news, it talks about three siblings (Jaxen, Tieler and Boston) that are all battling a rare white blood cell disease that makes food deadly. Each is allergic to pets, pollens and multiple foods, and has spent weeks at a time in the hospital — and they know they’ll be back sooner …
At first glance, a SAAS model may appear to be a solid option to streamline your workflow. But, you need to be sure you’re comparing apples to apples when it comes to the SAAS guys and us– does the SAAS model have all the features that the software solutions has and if not, are the missing features important to you? …
On Tuesday July 22nd we will be hosting a bake sale at ExecuTrain of Long Island. 100% of the proceeds from our sale will be donated to NORD (National Organization for Rare Diseases). Suggested donation of $1 per cookie. If you cannot attend our bake sale, and would still like to contribute to NORD, please feel free to make a donation …
NORD’s Director of the Scientific Advisory Committee, Dr. Marshall Summar, presented nine recommendations to the Energy & Commerce Committee at their hearing on the 21st Century Cures: Incorporating the Patients’ Perspective to accelerate the pace of medical breakthroughs and cures in the United States, and to speed up the process to get safe and effective drugs approved: Establish a Commission and National Plan to Determine …
Throughout our extensive research, we came across NORD’s (the National Organization for Rare Disorders) website countless time. They are not only a non-profit organization, but a provider of various information pertaining to the rare disease community. While their website may be overwhelming at first, it is filled with helpful information for all, whether you’re a patient, advocate, or medical professional. They …
As a previous Spartan Racer myself, we’re proud to say that we’re participating in the “CHARITY CHALLENGE” Spartan Sprint located in Killington, Vermont on September 21st 2014! With mental and physical challenges like no other, we’ll be running up mountainous hills for 4.5 miles that are over 3x the size of the Empire State Building, climbing our way over 10 …
What’s better than participating in charity events for the rare disease community and keeping our team healthy? Not much — which is why we have 5ks, walks, and other runs lined up this summer/fall! While the majority of the action in the rare disease community is either on the west coast in California or up north in Massachusetts, we were able to search far …
