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…
Patient Worthy collaborator Heal Canada has recently released the 5th edition of its digital magazine E³ Advocacy---Empower, Engage, Educate. This publication is dedicated to sharing articles, reflections, news, and resources…
Researchers discovered a version of the Cas protein that fits into adeno-associated viruses (AAVs). This virus can be engineered to deliver targeted therapy to cells and is a common approach…
Written by Jennifer, a patient living with WHIM syndrome. ### I’m in my sixties - but it wasn’t until a decade ago that I learned the name of the rare…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more. Jonathan…
By: Harsha K Rajasimha, Ph.D., Founder and CEO of Jeeva Clinical Trials Challenges Unique to Cell and Gene Therapies (CGTx) Clinical Trials Cell and gene therapies (CGTx) have emerged as…
On July 11th Dr. Mathew Winton went to Capitol Hill with senators and staff from both parties to discuss collaboration with the FDA regarding the pending termination of the Priority…
In unedited, early-access research published in Nature, a team of researchers explain that an estimated 60% of people with neurodevelopmental disorders remain undiagnosed, despite comprehensive genetic testing options. In many cases, this is…
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