The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SAR402663, Sanofi’s investigational gene therapy for neovascular age-related macular degeneration (AMD). As seen in a report, this…
Coffee lovers have long celebrated their favorite beverage for its taste and energy boost, but recent research offers another compelling reason to savor that daily cup: coffee directly and substantially…
I came to the United States from the Philippines in June 2016, full of excitement and dreams. I was ready to build a new life, work hard, and create something…
An experimental gene therapy known as AMT-130 has become the first treatment to successfully slow the progression of Huntington’s disease, marking a potential breakthrough in the fight against neurodegenerative disorders.…
Lexeo Therapeutics announced a significant regulatory development this week: the U.S. Food and Drug Administration (FDA) is open to a faster approval pathway for the company’s promising gene therapy, LX2006,…
Editor's Note: Patient Worthy is proud to share this story from our friends at the Glanzmann's Research Foundation (GRF). To see the article in its original format, please click here.…
Your story has the power to educate, motivate, and make a real difference in the lives of others. We are looking for patients with Metabolic Dysfunction-Associated Steatohepatitis, or MASH –…
Your story has the power to educate, motivate, and make a real difference in the lives of others. We are looking for patients with Metabolic Dysfunction-Associated Steatohepatitis, or MASH –…
In a groundbreaking international effort led by Dr. Shady Rahayel of Université de Montréal and reported by MSN.com, researchers have discovered new ways to predict which individuals with isolated REM…
AstraZeneca and Daiichi Sankyo have announced promising Phase 3 trial results for Datroway, their investigational antibody-drug conjugate (ADC) targeting triple-negative breast cancer (TNBC). The trial demonstrated that Datroway significantly slowed…
Bayer AG and its subsidiary BlueRock Therapeutics LP have announced encouraging 36-month follow-up data from the Phase I exPDite clinical trial of bemdaneprocel (BRT-DA01), an investigational cell therapy aimed at…
Editor's Note: Patient Worthy is proud to bring you this story from our friends at the Glanzmann Research Foundation. To see the article in its original format, please click here.…
Clinical trials often begin with optimism, sites are launched, projections look promising, and timelines are set. But when enrollment stalls, sponsors frequently call-in patient recruitment partners as a last-minute “rescue”…
Editor's Note: Patient Worthy is honored to share this story from our friends at the GRF (Glanzman's Research Foundation). To see the article in its original form, please click here.…
Doctors and researchers at the University of Colorado Anschutz Medical Campus have achieved a groundbreaking milestone by employing advanced Neuropixels technology in the study of Parkinson’s disease. Reported by Pharmabiz.com,…
Autoimmune diseases affect an estimated 400 to 600 million people globally, posing a growing challenge for healthcare systems and patients alike. Traditionally, treatment has focused on symptom management through broad…
Editor's Note: Patient Worthy is proud to bring you this story-share, originally written by Gwen Pike, founder of the podcast Empowerment in Illness. When I was in my twenties, life was…
A new study published in Aging-US reveals that Parkinson’s disease (PD) patients diagnosed in their 80s are significantly more likely to exhibit amyloid-beta buildup, which is an early marker of…
In a significant advancement for rare disease treatment in Japan, the Ministry of Health, Labour and Welfare (MHLW) has approved Bylvay® (odevixibat) for the management of pruritus associated with progressive…
When a woman hears the words, “You have cancer,” her world shifts in an instant. The journey ahead—marked by treatment, uncertainty, and emotional upheaval—can feel isolating, even for the strongest…
Each year, Patient Worthy covers the American College of Medical Genetics conference, the leading source of both genetic education for health care professionals and a leading source of new information…
As 2025 winds down, the pharmaceutical industry is bracing for a series of pivotal FDA decisions that could redefine treatment paradigms and reshape competitive dynamics across several major therapeutic areas.…
In a landmark step for personalized cancer care, the National Comprehensive Cancer Network® (NCCN®) has launched the NCCN Guidelines Navigator™—a cutting-edge digital platform that redefines how clinicians access and use…
Editor's Note: Patient Worthy is honored to bring you this patient story-share from Michael Grivas. For more information and resources, please visit the SADS Foundation. My name is Michael Grivas,…
In a quiet corner of Texas, Jenna Hornbuckle is living a story so medically rare, doctors estimate its probability in the trillionth range. A single mother of two, Jenna is…
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