HepaRegeniX GmbH has announced the dosing of the first patient in its Phase Ib clinical trial of HRX-215, a novel small-molecule inhibitor designed to promote liver regeneration. This marks a…
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…
In 2019 I presented with dark purple spots on my legs, and my neurologist decided to do lab work. My labs showed a high IGM level, so I was then…
Disclaimer: The views and opinions expressed in this article are those of the author and do not necessarily reflect the official policy or position of Patient Worthy. At just 5…
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.…
My symptoms began in 2001 and within 6 months I went from functionally uncomfortable to disabled to the point that I could barely lift myself out of bed because more…
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,…
Editor's Note: This is the first part of an ongoing story. The second part will be published Tuesday, June 17th. The fluorescent lights of the hospital room hummed, a stark…
Prader-Willi Syndrome (PWS) is a complex, rare genetic disorder known for its hallmark features: insatiable hunger, intellectual and behavioral challenges, and a host of physical health concerns. For families and…
Researchers at the UK Dementia Institute in London (UK DRI) have identified a common denominator for various gene mutations that cause amyotrophic lateral sclerosis (ALS). A new CRISPR study shed…
Living with a rare or chronic illness is a journey marked by uncertainty, resilience, and the power of community. For the last 10 years at Patient Worthy, individuals from all…
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…
Many people with rare diseases struggle to access personalized therapies simply because they lack a confirmed genetic diagnosis. This barrier blocks access to targeted treatments and clinical trials, leaving families…
Applied Therapeutics has encountered a significant setback in its quest to bring a new treatment to patients with a rare metabolic disorder. According to Biospace.com, the company announced that its…
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…
As many Patient Worthy readers are aware, Jenny Decker, a Charcot Marie Tooth patient, has made a serious commitment to circumnavigate the globe solo despite an incurable and eventually fatal…
We're honored to bring you this video, shared with us by Danny Gereg, that details some of his journey living with bladder cancer. Danny has stayed resilient and positive, finding…
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…
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