There are many conditions which can be cured or at least ameliorated with a stem cell transplant or an organ transplant. But even when the right donor is available, and…
How one deals with adversity is very unique to the situation and the individual. I have faced great adversity at different times in my life, some of which I handled…
, organized by the EveryLife Foundation for Rare Diseases, is one of the most important annual advocacy events for the rare disease patient community. The event is held the last…
Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which donor immune cells attack the recipient's tissues. The graft cells recognize…
In February 2024, Jacob Bell reported in Biopharma Dive that pharmaceutical company Takeda Pharmaceuticals is planning on rapidly advancing late-stage testing of TAK-861 for people with type 1 narcolepsy (also…
In 2020, Chris and Susan Finazzo received news that would change the trajectory of their lives. They learned that their sons Chase (now 9) and Dylan (now 6) had a…
In early March 2024, Clinical Trials Arena reports that global biopharmaceutical company Bristol Myers Squibb shared new data from the Phase 3 DAYBREAK study. Within the study, researchers explored how…
According to a story from Medscape, a new set of guidelines from the Kidney Disease: Improving Global Outcomes (KDIGO) aims to improve and update standards of care for antineutrophilic cytoplasmic…
In the rare disease community, fundraising plays a pivotal role in driving forward research and treatment initiatives, offering vital support to affected families, and guaranteeing access to necessary resources and…
Corticosteroids are one of the standards-of-care for treating alopecia areata, alongside treatments such as topical sensitizers and Rogaine. However, none of these treatments have a high level of evidence in…
If you have a health condition where you are misunderstood and perhaps judged by others for how you live your life to battle your symptoms, I invite you to read…
Children with late-infantile onset Batten disease (CLN2) may be treated with an enzyme replacement therapy (ERT). However, this therapy cannot reverse the damage or progression children have already experienced. Researchers…
According to a story from BioSpace, the current standard approaches for drug development aren't well suited for rare diseases, which have unique characteristics in comparison to more widespread illnesses. As…
At the end of January 2024, global healthcare leader Eli Lilly and Company shared that its therapy OLUMIANT (baricitinib) received regulatory approval in Canada for the treatment of severe alopecia…
The 20th annual WORLDSymposium took place from February 4-9, 2024. During the course of the research conference, at least one presentation centered on data from the Phase 2 Mini-COMET long-term…
American Journal of Cardiology Source: Association Between Atrial Fibrillation Symptoms and Clinical Outcomes: A Prospective Multicenter Registry Study
Having a chronic illness can be isolating. It can be difficult to share vulnerable and sensitive information about yourself with others, or to find ways to manage the trauma and…
People living with blood disorders like sickle cell disease or thalassemia will now have access to a new blood test that will reduce transfusion side effects. England’s National Health Service…
Cooper Meshew was diagnosed with Charcot-Marie-Tooth (CMT) disease at a very young age. The disorder affects one person in about 2,500 individuals. In fact, Cooper has an extremely rare variant…
Note: This patient story was contributed by one of Patient Worthy’s partners, HAE Junior – an organization dedicated to improving the lives of children and young people living with hereditary…
Written by Diane Wilkie My hATTR story began in the mid-1960’s when my father, a healthy, strong, outdoorsy guy in his late 40’s started tripping, experiencing numbness, and having dizzy…
Contributed by Scott Gray. Scott Gray is the co-founder and CEO of Clincierge, a provider of patient support services for clinical trials. Since 2015, Clincierge patient coordinators have managed logistics and reimbursements in…
Introduction I am a 50-year-old woman from the UK who has been suffering with an undiagnosed condition for 11-years for which I am seeking a diagnosis and medical treatment. I…
According to information in a recent press release from Wave Life Sciences, dosing has begun in a clinical trial investigating the RNA editing candidate, WVE-006. The drug treats alpha-1…
For years, scientists have been working on a completely new concept that generates new neurons in mice with Huntington's disease. They were able to demonstrate that the new cells could…
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