In a moving story of resilience and hope, a 10-year-old patient is embracing a new lease on life after receiving a heart transplant, seen as a medical miracle that has…
Not everyone who stands up for children’s health in Washington, DC, wears a suit or carries a briefcase. Sometimes, the most powerful advocates are the ones who have experienced the…
Global biotechnology leader CSL recently announced the FDA’s approval of ANDEMBRY, a monthly subcutaneous (under-the-skin) self-injection. The treatment was recently FDA approved for the prevention of hereditary angioedema (HAE) attacks…
LA Jolla Institute (LJI) scientists have recently discovered that T-cells, known primarily as disease fighters, may also play a role in the development of Parkinson’s disease. The researchers, working in…
Cure Rare Disease (CRD), a pioneering nonprofit biotechnology organization, has announced the addition of new scientific and strategic leaders, marking a significant step forward in its mission to develop therapies…
A recent article from Free Malaysia Today tells the remarkable story of Shu Hua, a woman who has not only surpassed a dire prognosis but continues to inspire others with…
A new study published at ScienceDirect.com sheds light on the profound social challenges faced by individuals and families living with rare diseases. The article examines how rare diseases, while medically…
A recent article from PMLiVE underscores the critical role of collaboration in advancing innovation and improving access to treatments for rare diseases. With over 7,000 rare diseases affecting millions of…
A recent article from The Economic Times spotlights the intriguing case of a young boy from South India who repeatedly fainted during baths—a perplexing pattern that led to the identification…
A remarkable story shared by Sportskeeda illustrates how a moment of frustration on the football field turned into hope for a family in need. In March 2021, after a controversial…
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…
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