As an official Friend of Rare Disease Week, we are highlighting some of our heroes and rare disease warriors' stories. Below was submitted by the ADNP Kids Research Foundation and…
It's no longer Valentines Day, but we're still sending love to the rare patient community! It's been an exciting week for Hemophilia B patients waiting for a new treatment! We…
Read Part 1 of Amy's Acromegaly Story here. The doctors told me that they felt the surgery removing the tumor was successful, but because of the position and size of…
February 16, 2018 Governor Herbert Declarations: Rare Disease Day on February 28th Undiagnosed Rare Disease Day on April 29th Park City, Utah. (February 16, 2018) – Rare and Undiagnosed Network…
Happy Friday, Patient Worthians! This week, we have a video from a PKU patient telling a first-hand account of the deterioration and serious consequences he faced after he went astray…
Nothing prepares families for the myriad of tests with strange sounding names that are required when they start to look for solutions to the problems brought about by a member's…
Andy Trapp is a videographer who runs AB Trapp Productions, a small business documenting weddings and family occasions. He has been living with uncontrolled PKU since the age of 8.…
Easton was born at thirty-six weeks gestation on January 6, 2017. He was almost immediately taken to the NICU for something called PPHN (Persistent Pulmomary Hypertension), Easton was intubated for…
Andy Trapp is a videographer who runs AB Trapp Productions, a small business documenting weddings and family occasions. He has been living with uncontrolled PKU since the age of 8.…
My name is Amy, and I’m a 37 year old single mom of two beautiful girls, ages 8 and 13. I work full time as a business analyst for a…
Welcome back, Patient Worthians! This week, we have an opportunity for Parkinson's patients, and an update on an Amyloidosis app. We also have two stories of teenagers whose lives suddenly…
Today, Professor Elsa Shapiro and Dr. Christina Lampe presented "Cognitive Impairment in Patients with MPS II: From Disease Burden to Cognitive Testing" at Satellite Symposium, an event sponsored by Shire…
A recent study from Michigan State University confirms that mice do indeed have significant similarities to humans which is beneficial for cancer research, reported News Wise. This has been in…
Yesterday, a group of experts presented "Changing lives: Long-term outcomes of MPS IVA & VI patients" at the Satellite Symposium sponsored by BioMarin. The program began with insights on the…
Happy Groundhog Day, Patient Worthians! It might not feel like it, but spring is its way! This week, we have a story of a family who copes with the challenges…
Happy Friday Patient Worthians! Believe it or not, we've almost made it through the first month of 2018! This week, we have a story of a young girl with Sanfillipo…
TGIF, Patient Worthians! This week, kids went to bed wearing inside out pajamas and wishing for snow. Their wishes came true-- and so did the wishes of many rare disease…
I know you’re fighting hard//I see your battle scars It’s clear you’ve come so far//You’ll grow stronger Alone, these words may feel like just expressions of encouragement; a…
Happy Friday, Patient Worthians! This week we have articles about people working together on different types of rare disease teams. We have stories from a family who supports two children…
When you are in the midst of difficult times in your life, what do you do to make it through? Do you try and find a way to hide out…
Patient Worthy recieved a note from CMT Warrior and Patient Worthy Contributor Quentin Martin of a worthwhile cause, that we think all our readers should know about! Quentin writes: If…
WASHINGTON, D.C. & SAN DIEGO — Children’s National Health System and Retrophin, Inc. (NASDAQ: RTRX) are pleased to announce the creation of the Retrophin Rare Disease Network at Children’s National.…
Welcome back, Patient Worthians! We're starting out the new year with stories of strength and awareness. This week, we have an article about a man with CSF leak, whose life…
Good news for Prometic Life Science new drug application! The pharmaceutical company has been searching for a cure for idiopathic pulmonary fibrosis (IPF), a rare lung disease which you can…
Happy New Years, Patient Worthians! New year, new you... new treatment news? This week, we have an article about a molecule that could change the way we treat cystic fibrosis.…
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