Orkambi (ivacaftor/lumacaftor) is the second precision medicine to be licensed for use in the UK by people with cystic fibrosis. The catch? It is not currently provided by the National…
Welcome to Friday Patient Worthians! This week we have stories on cystinosis and super moms with sickle cell! Additionally, we have a patient story on dystonia and an upcoming event. Lastly, an…
Welcome to Friday Patient Worthians! This week we have a PW Contribution on one woman's battle with Stiff Person's Syndrome. We also have a piece on using fear to serve you in…
The below article was originally published on May 31st, 2017 on the International Pemphigus and Pemphigoid Foundation website, and is being republished here with permission. It wasn’t easy getting to…
Want some more news, events and announcements on Sickle Cell? We got 'em! [one_half] [/one_half] [one_half_last] Brazilian Researchers Propose Classification of 3rd Sickle Cell Subtype SAFE Treatment for…
Happy Memorial Day Weekend Patient Worthians! This week we have an inspiring interview with the CEO and Founder of the Hereditary Neuropathy Foundation. We also have a motivating story on a cheerleader battling Friedreich's…
Want some more news, events and announcements on SCID? We got 'em! [one_half] [/one_half] [one_half_last] The Expense of Scientific Progress: The Price of Gene Therapy for SCID The…
Rare Disease patients tend to be tremendously aware of every aspect of our lives; our under-treated diseases, our lack of social support and our own coping mechanisms. We need more…
Want some more news, events and announcements on Beta Thalassemia? We got 'em! [one_half] [/one_half] [one_half_last] Atherosclerwhat? Pay Attention to Your Arteries if You Have Thalassemia Iron Supplements…
Want some more news, events and announcements on CGD? We got 'em! [one_half] [/one_half] [one_half_last] Testing New CGD Treatments Just Got Easier [/one_half_last] [one_half] [/one_half] [one_half_last] The Most…
Positive Thoughts Kim Frederickson was diagnosed with pulmonary fibrosis (PF) in 2014. She has used her skills as an author to share her story. Her motto is to focus on…
Happy Friday Patient Worthians! This week we have two awesome contributions. One addresses grieving as a parent of a child with rare disease. Another addresses living with CRPS and other…
I felt the cold sweat racing down my back. I felt like I was drowning, but there was no water around. By this point in my life, I had experienced…
Welcome back to Editor's Choice! May is Lyme Disease Awareness Month, and to bring you awareness we have a 7-minute documentary you won't want to miss! We also have some…
There is no way to prepare yourself to hear that your child has an incurable rare disease. After the initial shock of learning that their child will most likely die…
I was discussing with my sister’s husband a presentation on what to do when diagnosed with a chronic illness. I was going to present this to People with Parkinson’s (PwP),…
"I have chronic Lyme Disease and it's chronic because it took so long to get it treated." William and Mary student Alexis Plofchan is the subject of a seven-minute documentary directed by…
Happy Friday Patient Worthians! You might notice that the headline has a lot of acronyms in it... ah, welcome to the rare disease world! We DO have answers for what all of that…
National Center for Advancing Translational Sciences (NCATS) is a division of the NIH, located in Bethesda MD. On Friday June 30th they will have an all day program to discuss…
The drug, Ceriliponase alfa from BioMarin, helped in a condition that has led inexorably to disability and death: Batten Disease – specifically CLN2. Families of kids facing this prognosis are…
Happy Friday Patient Worthians! Did you know it was World PI Week this week? We have a post on the role pets have with PI. We also have a story on a…
This article was originally published on Luke's blog and is shared here with permission. Keeping our kids safe is every parent’s number one job. Even as an adult I associate…
Happy Friday Patient Worthians! PW Contributor Tom Seaman talks about what it's like to overcome the mental toll of your rare disease diagnosis. Another PW Contributor suffering from TN, dysautonomia and…
The following blog post was written on October 4th, 2016 by a #raredad whose daughter is battling a chronic illness. With his permission, we are republishing this powerful memory here.…
Ever wonder what it’s like to be so sick and disabled that you can’t live a normal life but you aren’t sick or disabled enough to actually be considered disabled?…
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