Editor's Note: Patient Worthy is honored to share this story from our friends at Elephants & Tea. To see the original article, please click here. One day, many years ago,…
An estimated 47% of infants have some form of plagiocephaly (a flat spot on the head), and up to 10% will eventually need a corrective helmet to help reshape their skull. Some…
Assertio Holdings has announced the results of the first real-world evidence study evaluating patient experience with SYMPAZAN® (clobazam) oral film, presented at the 150th Annual Meeting of the American Neurological…
In the spring of 2013, as a Creative Writing Major, for my senior thesis I got to write any type of creative piece of my choosing. I chose to write…
aTyr Pharma suffered a major setback on September 15, 2025, as its stock price plummeted more than 80% following disappointing results from a pivotal Phase 3 trial of its lead…
In a breakthrough study, scientists have uncovered a surprising ally in the fight against cancer: the bacteria living within tumors. According to World Pharma News, an international team led by…
A new study conducted at the Huntsman Cancer Institute in Utah suggests that exercise has a beneficial effect on the body, such as reprogramming, that alters the gene’s function, identity…
A newly developed assessment method that brings together a blood-based measurement and a physical body metric is showing promise for forecasting both survival chances and nutritional health in patients battling…
Medetoidine is an animal sedative increasingly being found in human opioid deaths according to published reports in the journal “The Conversation” and shared on MedicalXpress. Synthetic opioids claim thousands of lives…
Dewpoint Therapeutics, a U.S.-based biotechnology innovator, has announced the successful closure of its Series D financing round. As reported by thepharmaletter, Dewpoint Therapeutics is renowned for its pioneering work in…
Editor's Note: Patient Worthy is proud to share this story from our friends at No Stomach for Cancer. To see the article in its original form, please click here. In…
Life has a way of catching us off guard when we least expect it. For me, it all started with severe stomach pains that led to an unexpected hospital visit.…
Palvella Therapeutics, Inc. has announced a key milestone in the development of therapies for rare genetic skin diseases: the completion of enrollment in its Phase 2 TOIVA trial of QTORIN™…
Genetic testing is now more available than ever before. For those who suspect a genetic disorder, the best route is often through a genetic counselor. They can advise you not…
In our third installment from our series of reports provided by our partner TREND Community, we are bringing you several reports that analyze the effects of sleep disorders. This week…
Health Canada’s recent approval of adjuvant (applied after surgery) Keytruda (pembrolizumab) in high-risk invasive cancer of the bladder saw patients remain free of cancer in double the current standard, according…
Editor's Note: This article was shared with Patient Worthy by our friends at PDSA.org. To see the article in its original format, please click here. I had heard of lupus…
Capsida Biotherapeutics has suspended a clinical trial of its experimental gene therapy after the first child to receive the treatment died, sending shockwaves through the rare disease and gene therapy…
Glioblastoma is notorious as the most lethal form of malignant brain tumor, with most patients surviving just one or two years after diagnosis. Part of what makes these tumors so…
Acknowledgement: Patient Worthy is honored to share this story from our friends at GACI Global. To see the article in its original format, please click here. Born on World Heart…
A recent milestone in gene therapy is transforming the outlook for people with rare genetic skin disorders, signaling a new era of personalized medicine and hope for patients who previously…
Research into rare diseases has long faced obstacles, from limited patient numbers to the challenge of replicating disease biology in the lab. A new wave of innovation, highlighted in the…
The U.S. Food and Drug Administration (FDA) has introduced a new regulatory framework aimed at improving and clarifying the approval process for drugs targeting rare diseases, also known as orphan…
Acknowledgement: Patient Worthy is proud to share this article from our friends at Elephants & Tea. To see the article in its original form, please click here. I wish someone…
The U.S. Food and Drug Administration (FDA) has granted an expanded indication for Vonvendi, Takeda’s recombinant von Willebrand factor (rVWF) therapy, marking a significant advancement in the management of rare…
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