Check out part 1 of this LGS post here. After prepping: Arrive at the airport early – Ask the airline agent at check-in about getting your wheelchair assistance. Use this…
Spinal muscular atrophy (SMA) is the leading genetic cause of infant mortality in the USA. So why aren't hospitals in Texas screening newborns for it? That's what a group of…
Traveling is certainly possible and can be more relaxing with some pre-planning and organization. Some of these ideas may not be applicable to you and your family. I have compiled…
There's a new treatment for X-Linked Hypophosphatemia (XLH) that may be available in the not-so-distant future. XLH occurs when the kidneys process phosphate abnormally, leading to excess phosphate loss in…
Happy Friday Patient Worthians! This week, we some news to be happy about! We have an awesome story of how thinking like a start up will help lead to more…
Two-year-old Max Payne from Immingham, England, holds onto the yellow rail of his play pen as he stands up. He glances up at the video camera for a moment, and then…
Date of issue: October 19, 2017 Boxing legend Michael Carruth was today firmly in Cystic Fibrosis Ireland's corner at Dublin's Irish Film Institute for the launch of a new awareness…
Sherry Jo Ward sits in an orange armchair, playing harmonica in the center of the stage. She leans forward on the matching ottoman for emphasis. Her walker is never out…
Rebecca Wanosik was already a pro when it came to being a mom. Zedyn was her fifth child. She knew to trust her gut when, three weeks after her baby…
Rare disease research can feel like an uphill battle. First of all, rare diseases have a harder time receiving funding. After that, it takes months or even years to have…
Happy Friday Patient Worthians! This week, we have an awesome story about PW Partner Avery's Angels. We also have a PW Contribution about key lessons everyone can learn from the…
Sickle cell anemia results from a single mutation in the hemoglobin gene. The mutation creates a protein which has an atypical structure. This altered structure throws a wrench in the…
We've written about CRISPR technology before, and it's potential for treating sickle cell anemia. But in a latest study done at the University of California, Berkeley, sponsored by the NIH,…
TGIF Patient Worthians! This week, we have two opportunities where you can spread some love. You can help a young boy with microcephaly and/or contribute to amyloidosis research. We also…
October 21, 2017 Remember this deadline if you have a alpha or beta thalassemia, or another significant form of thalassemia, and are seeking an advanced degree in the US or abroad…
It's Fall "Y'all"! This week, we have PW Contribution from a man with CMT. We also have one on raising a child with rare disease. We also have good news…
HSG 2017 is coming your way Denver! The Huntington Study Group (HSG) is a global professional group dedicated to finding better treatments and ultimately a cure for Huntington Disease (HD). They…
Pack your bags LGS Warriors, your conference is headed to Orlando, FL this November! The LGS Foundation is holding it's fifth International Family and Professional Conference to promote collaboration between LGS…
PW Partner the Amyloidosis Foundation (AF) is hosting their annual amyloidosis research benefit and you won't want to miss it! The AF was founded by two amazing amyloidosis patients who strived…
This year, The International Advocate for Glycoprotein Storage Diseases (ISMRD) has moved their International Conference from the US to Rome, Italy! The ISMRD is a non-profit who aims to be…
A recent report from the Food & Drug Administration reveals renewed efforts for the treatment of one of the rarest forms of non-Hodgkin's Lymphoma, Mantle Cell Lymphoma. Due to promising advancement…
The first story that comes to Quentin's mind about a turning point in his life is about his attendance at a school for the blind. When Quentin lost his vision…
I like nonsense, it wakes up the brain cells. Fantasy is a necessary ingredient in living, It’s a way of looking at life through the wrong end of a telescope.…
Welcome back Patient Worthians! We are back this week from the 2017 Global Genes Rare Advocacy Summit. Did you attend? If so, what did you learn? Let us know here. This…
The Pulmonary Fibrosis Foundation (PFF) is hosting their biennial summit this November! Pulmonary fibrosis covers 200 different conditions that include sarcoidosis, IPF, scleroderma and other rare diseases. The conditions are characterized by…
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