A recent article from Pharmaceutical Manufacturer highlights a significant achievement in the rare disease sector, as Rare Disease Biotech announced reaching a major milestone in the development of its new…
A recent article published in the Orphanet Journal of Rare Diseases highlights the launch and goals of RARE-Impact, a new European project designed to enhance the diagnosis, care, and overall…
Researchers have discovered a potential “window” whereby babies born with inherited diseases may be treated with gene therapy sent directly into their circulatory systems. The window consists of circulating stem…
Launched in May 2015, Patient Worthy set out with a bold mission: to amplify the voices of rare disease and chronic illness patients, and to connect and inform a community…
A recent study has revealed that vitamin B3 could offer hope for patients suffering from a rare genetic disorder that causes accelerated aging. As reported by ScienceBlog, researchers found that…
A dedicated Hull resident, Jonny Wightman, is preparing to cycle an impressive 100 miles to raise awareness and funds for rare diseases. As reported by Hull Daily Mail, Jonny’s motivation…
The FDA recently informed Muscular Dystrophy News that it has granted orphan drug designation to the experimental drug ISX9-CPC, a product of the IPS Heart company located in Houston, Texas.…
Pearl Jam front-man Eddie Vedder takes center stage in the new documentary “Matter of Time,” offering fans and viewers a rare, intimate glimpse into his life as both an artist…
In 2014 the U.S. experienced a nationwide outbreak of EV-D68, raising concerns about children’s respiratory health. Acute flaccid myelitis disease (AFM) also spiked. EV-D68 is defined as a group of…
The FDA is taking significant steps to update and streamline its regulatory approach to rare diseases, according to a recent article from BioCentury.com. Recognizing the unique challenges faced by patients,…
Alnylam Pharmaceuticals has launched a unique promotional campaign for Givlaari, its treatment for acute hepatic porphyria (AHP), that swaps the usual clinical narratives for something more artistic: dance. As reported…
In a significant stride for global health, the United Nations recently adopted a historic resolution dedicated to rare diseases. This move, as reported by Medics 4 Rare Diseases, celebrated by…
In a groundbreaking medical achievement, physicians have administered the world’s first personalized gene-editing therapy to an infant suffering from a rare and fatal genetic disorder. The innovative treatment, described at…
Three hundred and ten: The number of days we had prior to our lives forever changing. On April 13, 2022, it was a day like any other. My husband and…
In the realm of rare diseases, effective communication can be a formidable challenge, especially when symptoms impact speech and expression. Remembering our very first post on May 19, 2015, Patient…
Rebecca Hind, a woman from the UK, endured a life-altering ordeal after what she thought was a simple case of food poisoning turned out to be an extremely rare and…
In the remote town of Serrinha dos Pintos in northeastern Brazil, a rare genetic disorder once shrouded in mystery has shaped the lives of many families for generations. As reported…
"Rock Bottom" is the story of a family who’s second born child, a son named Ashton, was born with Mucolipidosis Type II, a disease so rare and so frequently mistaken…
Editor's Note: Patient Worthy is grateful to present this article that was written by and shared with permission from Sue Skavlem, a care partner to her husband who is living with…
Part 3 - Our Post-Transplant Journey (the first year) Continued from Part 2 From the nurses and doctors to the therapists and social workers and everybody else involved in Becky’s…
This article was written by and shared with permission from Shannon Wieloch, MS. Shannon is a certified genetic counselor and patient advocate for the Aicardi-Goutiere Syndrome Advocacy Association and founder of Stork Genetics.…
In an exciting and heartwarming shift towards inclusivity, the world of sports is embracing a new era of diversity—one where disabled athletes are not only welcomed but celebrated for their…
This article was written by and shared with permission from Shannon Wieloch, MS. Shannon is a certified genetic counselor and patient advocate for the Aicardi-Goutiere Syndrome Advocacy Association and founder of Stork Genetics.…
Meet Liam, a 12-year-old patient at Shriners Children’s St. Louis, born with myelomeningocele, a severe form of spina bifida. This condition affects sensation in his legs, leaving him without feeling…
Written by Heather Shorten, Founder, Pompe Alliance On July 15th, 2015, I was diagnosed with Pompe disease after about 3 years of searching for a diagnosis. I know 3 years…
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