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
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
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
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
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,
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
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
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
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
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
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
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
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
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
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
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
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
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
Coalition to Cure CHD2 THIS MONDAY!!!💬 Join CCC for our Monthly Meetup on Monday, June 1st 12 PM Central Time (1 PM ET | 6 PM UK | 7 PM Central Europe)!
Spend an hour on Zoom with fellow CHD2 parents, family members, and caregivers where you can ask questions and share your glows and grows about your loved ones affected by CHD2-related disorders.
You’ll also have a chance to learn about the latest research and ask questions about how it could help your family.
Register here: bit.ly/CCC-Monthly-Meetup
What topic would you like to discuss this month?
#CHD2 #CureCHD2 #StrongerTogether
bit.ly
A philosophy lesson on caregiver anxiety, doom scrolling, and learning to live with uncertainty through curiosity, presence, and openness.
@AmericanLiverFoundationA major breakthrough in liver health: the FDA has approved the first treatment for chronic hepatitis delta virus (HDV).
This milestone marks an important step forward in care and hope for patients and families navigating HDV.
👉 Read more: alf.social/FDA-HDV
#HDV #LiverHealth #Hepatitis #LiverDisease
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