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…
ACKNOWLEDGEMENT – Patient Worthy is honored to partner with The Fatty Liver Foundation to provide you with this article. The Fatty Liver Foundation aims to improve the diagnosis, treatment & support…
After more than a decade of development, families say time is running out for children with Barth syndrome as the experimental therapy elamipretide faces yet another regulatory delay. Reported by…
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…
When baby KJ was diagnosed with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency—a rare metabolic disorder that can trigger life-threatening ammonia buildup, his family faced a narrow path: strict dietary…
Acknowledgment: This story is sponsored by GSK and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted, and valuable…
The FDA has approved BridgeBio Pharma’s acoramidis, branded as Attruby, for transthyretin amyloidosis cardiomyopathy (ATTR-CM), positioning the oral therapy to compete head-to-head with Pfizer’s tafamidis franchise (Vyndamax/Vyndaqel/Vynmac). According to PharmaPhorum.com,…
A study co-led by researchers at The Hospital for Sick Children (SickKids) and the University of Toronto’s Temerty Faculty of Medicine sheds light on why some very-high-risk childhood brain tumors…
Transthyretin amyloid cardiomyopathy (ATTR-CM) is an infiltrative heart disease driven by protein instability. Normally a tetramer, transthyretin can dissociate into monomers that misfold, aggregate, and deposit as amyloid fibrils within…
Biopharma.com reported sharp contrasts, with a landmark approval for a rare disease therapy alongside a high-profile trial miss, and a series of corporate moves reflecting market access and financing realities.…
Studies by highly accredited Mayo Clinic and Barrow Neurological researchers report finding an association between living in close proximity to a golf course and an increased risk of Parkinson’s disease…
Diagnosing transthyretin amyloid cardiomyopathy (ATTR-CM) is challenging because its symptoms are vague and overlap with many conditions. Providers typically follow a stepwise approach that combines history-taking, targeted testing, and imaging…
Genetics: a cosmic role of the dice that plays a role in so many medical conditions, including bladder cancer. Mutations in FGFR3, PIK3CA, KDM6A, and TP53 are commonly associated with bladder cancer, but…
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…
Acknowledgment: This story is sponsored by Astellas Pharma US Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
When we are faced with a limiting health issue where performing “simple” everyday activities becomes a challenge, our self-confidence and self-worth can dwindle. This can happen with any health condition…
Researchers in Japan have developed a personalized mRNA vaccine that shows promise for treating recurrent gastric cancer, specifically peritoneal metastasis, which is often difficult to manage with existing therapies. This…
Shebli Atrash, MD, Levine Cancer Institute medical oncologist and associate professor of medicine at the University of Wake Forest interviewed with Medical Express following his presentation at the 2025 Conference in…
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…
Vepdegestrant, an investigational oral selective estrogen receptor degrader (SERD), has reached a critical milestone as the U.S. Food and Drug Administration (FDA) reviews its New Drug Application (NDA) for use…
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…
Recent advancements in the treatment of metastatic urothelial cancer (mUC) have dramatically improved patient outcomes, primarily due to the introduction of antibody-drug conjugates (ADCs) and immune checkpoint inhibitors (ICIs). According…
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