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…
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…
Stanford University has recently secured a prestigious grant from the U.S. National Institutes of Health (NIH) to accelerate the clinical development of Tiakis Biotech’s novel therapy, Elafin (also called Tiprelestat),…
Editor's Note: Patient Worthy is honored to share this article from our friends at Courageous Parents Network. Parents of well children regularly receive anticipatory guidance–proactive communication about what’s coming in…
Editor's Note: This article is an opinion piece shared with Patient Worthy from our partner network. Opinion pieces may not reflect the views of Patient Worthy, its employees, or any…
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…
Ireland has unveiled a new national strategy aimed at transforming the diagnosis and care of the estimated 300,000 people in the country living with rare diseases. According to The Irish…
After cancer treatment, many patients are left with a haunting uncertainty: Is it really gone—and will it stay that way? Even when scans appear clear, questions linger. Now, a newer…
A groundbreaking study from the Weizmann Institute of Science and Sheba Medical Center has brought renewed hope for children with kaposiform lymphangiomatosis (KLA), a rare and life-threatening genetic disorder of…
August 2025 was a landmark month for rare disease innovation, as the U.S. Food and Drug Administration (FDA) granted four historic approvals, each representing a first for their respective conditions,…
Baylor Genetics is set to showcase groundbreaking innovations in RNA sequencing for rare disease diagnosis at the 2025 Advances in Genome Biology and Technology (AGBT) Precision Health Meeting, according to…
A recent report from NDTV World brings attention to a rare but significant public health event: an American man has tested positive for plague, the infectious disease historically known for…
Health authorities in Queensland, Australia, have issued a public warning after the discovery of Naegleria fowleri—a rare but deadly “brain-eating” amoeba—in a town’s water supply. As reported by The Medical…
According to a recent article from JournalFeed, the relationship between type 2 diabetes and metabolic dysfunction-associated steatotic liver disease (MASLD) is increasingly coming to the forefront of clinical care. MASLD,…
In our third installment from our series of reports provided by our partner TREND Community, we are going to be focusing on a different condition over four weeks. This week…
A team of scientists from Rice University, Baylor College of Medicine, and the Jan and Dan Duncan Neurological Research Institute have introduced a significant breakthrough in the study of complex…
Early clinical data from a Phase I/II study at UMass Chan Medical School point to meaningful, measurable benefits from a central nervous system gene therapy for GM2 gangliosidosis, which encompasses…
A new study recently published in the European journal of Preventive Cardiology and reported by Medical News Today, noted that individuals who added extra steps plus reasonable speed to their…
According to an article in Inside Precision Medicine Researchers have developed a new, easy-to-use test called SHINE-TB that can quickly diagnose tuberculosis (TB) using saliva samples. This test is based…
In our second installment from our series of reports provided by our partner TREND Community, we are going to be focusing on a different condition over four weeks. This week…
Novartis has announced encouraging results from two pivotal studies involving its experimental drug, ianalumab, targeting two different autoimmune indications. As reported by BioPharma Dive, the Swiss pharmaceutical company revealed positive data…
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…
A recent study has unveiled a novel treatment that could significantly lessen brain damage following a stroke, offering hope for more effective therapies in the future. According to World Pharma…
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