Millions of social media posts and billions of views have turned TikTok into an influential platform for mental health conversations, but experts warn that using the app to self-diagnose can…
AstraZeneca, a global leader in pharmaceuticals, has taken a significant step into the rare disease arena in South Africa, aiming to improve diagnosis, treatment, and support for patients facing these…
Sanofi has announced a significant regulatory milestone for its investigational therapy SAR446523, a monoclonal antibody targeting GPRC5D, after the U.S. Food and Drug Administration (FDA) granted it orphan drug designation…
Australian athlete Tiana Death has bravely shared her journey after being struck by autoimmune encephalitis, a rare and serious brain disease that upended her life and forced her to relearn…
Biogen has achieved a significant regulatory milestone with the UK Medicines and Healthcare products Regulatory Agency (MHRA) granting approval for Qalsody (tofersen), a treatment specifically developed for a rare and…
Calidi Biotherapeutics has achieved a significant milestone with the U.S. Food and Drug Administration (FDA) granting Fast Track designation to its experimental therapy, CLD-201 SuperNova, for the treatment of glioblastoma.…
Acknowledgement: This article was originally written by Gillian Sapia, a mother of a child living with galactosemia, for Underground Rare Road. To see the original article, please click here. When…
“I have to be dying”, I said earnestly to my husband for the second time that year, “at the very least, I don’t think there is much more pain I…
A recent research article in ScienceDirect.com details a breakthrough in visualizing the rapid, complex movements of proteins using high-speed atomic force microscopy (HS-AFM). This cutting-edge imaging technology allows scientists to…
A recently published case report has shed light on an uncommon cause of abdominal pain in adults: small bowel intussusception due to a benign tumor known as a lipoma. As…
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…
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