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…
Patients are often caught in a system of impersonal treatment when the "business" of medicine and health insurance predominate over human health. It is extremely rare today for physicians to…
A new study by MIT researchers reveals new potential targets such as cellular pathways and genes that have not been previously linked to Alzheimer’s. These results may provide answers to…
Since its inception in 2015, PatientWorthy.com has emerged as a beacon of hope, information, and support for the global rare disease community. From humble beginnings with its very first post,…
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