According to a recent article on PharmaBiz, HAYA Therapeutics has announced the completion of dosing in the first cohort of its phase 1 clinical study evaluating HTX-001, an investigational RNA-targeting…
According to a recent article in The Manila Times, Jade Biosciences has begun dosing participants in a Phase 2 clinical study, known as JUNIPER, evaluating its investigational therapy JADE101 for…
An article from Medscape highlighted recent advances in treatment that are reshaping the clinical approach to epidermolysis bullosa (EB), transitioning care from largely supportive management to therapies that address genetic…
A recent article by BioPharmaDive highlighted a mid-stage clinical trial that evaluted an investigational Parkinson’s disease therapy co-developed by Biogen and Denali Therapeutics, and that failed to meet its primary…
Johnson & Johnson announced FDA approval of a supplemental new drug application for Caplyta (lumateperone) for the prevention of relapse in schizophrenia, based on compelling long-term clinical data and reported…
A growing body of research highlights the broader benefits of human papillomavirus (HPV) vaccination beyond its long-standing focus on cervical cancer prevention. A recent large-scale retrospective cohort study published in…
According to a recent article by The Manila Times, Design Therapeutics has reported encouraging interim results from its ongoing Phase 1/2 RESTORE-FA study, highlighting both clinical improvement and biomarker activity…
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)…
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