Go here to read Part 1, Part 2, and Part 3 of this series provided by TREND Community about Prader-Willi Syndrome. In January 2025, the TREND Community published another comprehensive report…
The treatment landscape for hemophilia B has taken a historic leap forward, as the first patient in the UK’s National Health Service (NHS) has received Hemgenix, a pioneering gene therapy…
Madrigal Pharmaceuticals has recently captured significant attention in the race to treat metabolic dysfunction-associated steatohepatitis (MASH), formerly known as nonalcoholic steatohepatitis (NASH). As the landscape of liver disease therapeutics evolves,…
As reported by HD Buzz, a significant step forward for Huntington’s disease (HD) research, Roche has announced that the first patient has been dosed in its new gene therapy clinical…
Anixa Biosciences has reached a new milestone in the development of its innovative CAR-T cell therapy, announcing the dosing of the first patient in the fourth cohort of its ongoing…
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…
A significant milestone has been reached in multiple sclerosis (MS) research with the imaging of the first patient in a clinical trial using a novel PET tracer. This innovative approach…
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…
The world of healthcare is changing rapidly, and technology is at the heart of its evolution. The latest innovation making waves is an AI-powered medical receptionist that’s not only efficient…
Patient Worthy is grateful to our partner Elephants & Tea for providing the following article by Laura DeKraker Lang-Ree, a cancer caregiver to her daughter Cecilia who survived childhood leukemia.…
Mama we are all different colors and we are all beautiful. For our final Throwback Thursday we bring to you the story of Mariana, a young girl diagnosed with Pfeiffer…
In a remarkable demonstration of medical innovation, a Florida-based surgeon recently performed a robotic surgery on a patient located over 7,000 miles away. This groundbreaking procedure highlights the immense potential…
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…
Go here to read Part 1 and Part 2 of this series provided by TREND Community about Prader-Willi Syndrome. TREND Community released a revealing report in November 2024 titled “Pulse…
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…
In the vast world of podcasts, “Wait, How Do You Spell That?” stands out as a beacon for patients, families, and advocates navigating the complex, often isolating journey of rare…
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 report from the Times of India tells the inspiring story of a serving Indian Army soldier who overcame a critical health battle thanks to a successful lung transplant…
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…
I had learned that the many twists and turns on our rare disease path were similar to the families we met Originally published on May 11, 2022, this article explores celebrates author…
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…
For Part 1 of this series provided to you by TREND Communities, please click here. On July 10, 2024, TREND Community released an impactful new report titled “Prader-Willi Syndrome: Perspectives…
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