Novartis and PTC Therapeutics have advanced their Huntington's disease candidate, votoplam, into Phase 3 development following promising mid-stage results. The decision reflects confidence in the program's potential while signaling caution…
As reported on MedicalXpress, a newly developed RNA-focused analytical method may improve the detection and characterization of repeat expansion disorders, a group of genetic conditions that includes Huntington’s disease, amyotrophic…
A recent article from The Manila Times highlighted findings from the ongoing IMPACT‑TD Registry underscore a persistent gap in diagnosing tardive dyskinesia (TD), particularly among younger adults with underlying mood…
BridgeBio's Attruby (acoramidis) is positioning itself as a formidable competitor in the transthyretin amyloid cardiomyopathy (ATTR-CM) market, with new pivotal trial data and indirect comparisons suggesting potential advantages over Pfizer's…
A recent article from SciTechDaily describes the promising approach instituted at a private, non-profit biomedical research firm in San Francisco that incorporates data science, AI, stem cell biology, CRISPR, and…
According to a recent press release, Artios Pharma has begun dosing patients in a global Phase 2 clinical trial evaluating its investigational DNA polymerase theta (Polθ) inhibitor, ART6043, in combination…
A significant advancement in antiviral therapy has emerged with the successful development of a CRISPR-Cas13d-based system designed to combat hepatitis E virus (HEV) infections. This innovative approach harnesses the precision…
In a recent press release from Pfizer, it was shared that the European Commission (EC) has broadened the approved use of Pfizer’s marstacimab (brand name HYMPAVZI), extending its indication to…
A recent report by the Manila Times highlighted Madrigal Pharmaceuticals' unveiling of a series of new analyses and real-world findings supporting the therapeutic profile of resmetirom (Rezdiffra) for metabolic dysfunction-associated…
UniQure has announced plans to submit its groundbreaking Huntington's disease gene therapy, AMT-130, for regulatory approval in the United Kingdom later this year, marking a significant step forward for the…
According to a recent report on MedTechDive, Guardant Health has received U.S. Food and Drug Administration (FDA) approval for an updated version of its liquid biopsy assay, marking a significant…
Editor's Note: Patient Worthy is honored to present this story by Heather Doyle, originally published by the United Porphyrias Association. To see the article in its original format, please click…
Editor's Note: This piece, originally written by Kayla DeSonier, was shared with us by our friends at Elephants & Tea. To see the article in its original format, please click…
Editor's Note: This article was shared with us by our friends at the Cure NF2 Foundation, in honor of May 22nd being World NF2 Awareness Day. To see the article…
Exelixis has taken a major step forward in its colorectal cancer development pipeline by securing supplies of Merck's Keytruda for an upcoming phase 3 clinical trial. As reported by Fierce…
Betty Vertin is a mother, writer and a fierce defender of the decisions she and her husband Jason made about the seven children they brought into this world. Betty writes…
According to the CDC, a look back at April 2026 cumulative reports showed more than 139 pediatric deaths with approximately 250,000 people hospitalized. In simple terms the 2025-2026 US flu…
Atossa Therapeutics Inc. has achieved a significant regulatory milestone with the U.S. Food and Drug Administration's grant of Rare Pediatric Disease (RPD) designation to (Z)-endoxifen for treating McCune-Albright syndrome (MAS)…
An article from University of Michigan highlighted Jolanta Grembecka and her husband Tomasz Cierpicki, both assistant professors in U of M's Department of Pathology, who have been working to restore…
Milenia Makani is an abstract artist living in London. She lives with Hypermobile Ehlers-Danlos Syndrome (hEDS), and has launched an international art fundraiser to support medical research through the Ehlers-Danlos…
What happens when a pharmaceutical first-mover discovers its historic achievement may not be a blockbuster? Arvinas and Pfizer found an answer this week by handing off their pioneering breast cancer…
What is Parkinson’s Disease – known as PD for short? Parkinson’s Disease was first discovered in 1817 by a British doctor named James Parkinson. From observations, he noticed 7 people…
A recent article by Dr. Rosalie Greenberg explores the possible relationship of infections caused by ticks and psychiatric conditions in children, including PANS or PANDA which are sudden onset. For…
Abeona Therapeutics Inc. has announced a significant expansion in access to ZEVASKYN, marking the activation of Children's Hospital of Philadelphia (CHOP) as the sixth Qualified Treatment Center for this groundbreaking…
As reported on PharmaBiz, Regenxbio has announced encouraging topline findings from the pivotal Phase III portion of its ongoing AFFINITY DUCHENNE clinical program evaluating RGX-202, an investigational gene therapy for…
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