Cystic Fibrosis
International Clinical Trials Day
Editor’s Note: This article was originally written and shared with us by Caleigh Haber. This day hits really close to home for me. I was
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A Deeper Dive into the Ebola Virus
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THE LATEST FROM PATIENT WORTHY
RSV
GSK’s Arexvy Gains Expanded Approval in Japan to Include Younger High-Risk Adults
As reported by PharmaBiz, Japan’s Ministry of Health, Labour and Welfare (MHLW) has broadened the approved use of GSK’s respiratory syncytial virus (RSV) vaccine, Arexvy,
Hypertrophic Cardiomyopathy
HAYA Therapeutics Advances First-in-Class RNA Therapy Into Phase 1 Trial for Cardiac Fibrosis
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
IgA Nephropathy (IgAN)
Jade Biosciences Initiates Phase 2 Trial of JADE101 in IgA Nephropathy
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
Epidermolysis Bullosa
Epidermolysis Bullosa Care Enters a New Era With Gene-Targeted and Wound-Healing Therapies
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
Parkinson's Disease
Biogen–Denali Parkinson’s Therapy Falters in Mid-Stage Trial, Shifting Focus to Targeted Subgroups
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
Schizophrenia
Caplyta Gains FDA Approval for Schizophrenia Relapse Prevention with Significant Clinical Benefit
Johnson & Johnson announced FDA approval of a supplemental new drug application for Caplyta (lumateperone) for the prevention of relapse in schizophrenia, based on compelling
Human papillomavirus
HPV Vaccination Linked to Significant Reduction in Cancer Risk Among Males
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
Friedreich ataxia
DT-216P2 Shows Early Clinical and Biomarker Gains in Friedreich Ataxia Trial
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
Huntington's Disease
Votoplam Advances to Phase 3: Novartis and PTC Chart Measured Path for Huntington’s Disease Treatment
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
Rare Disease
RNA “Origami” Nanopore Technique Shows Promise for Faster Diagnosis of Repeat Expansion Disorders
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
Tardive Dyskinesia
Low Diagnosis Rates Persist for Tardive Dyskinesia in Young Adults with Mood Disorders, Registry Data Show
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
Transthyretin amyloid cardiomyopathy
Attruby Emerges as a Compelling Alternative to Vyndamax in Cardiac Amyloidosis Treatment
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
Rare Disease
How A Rare Genetic Condition May Be Treated by A Simple Vitamin
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,
BRCA-mutated Breast Cancer
Artios Initiates Phase 2 Trial of Novel Polθ Inhibitor in BRCA-Mutated Breast Cancer
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
Hepatitis E
RNA-Targeting CRISPR: A Breakthrough Approach to Inhibiting Hepatitis E Virus
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
Hemophilia
European Commission Expands Approval of Pfizer’s Marstacimab for Hemophilia Patients With Inhibitors
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
Metabolic dysfunction-associated steatohepatitis
Madrigal Highlights Expanding Evidence for Resmetirom in MASH at EASL 2026
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
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A bereaved mother navigates grief after the death of her child, maintaining connection through writing letters to heaven - a spiritual message of love and...
The unofficial start of summer is here.
Time to book your trip to see fellow cystinosis warriors in the Windy City through Speak Up, Speak Out (closing soon!)
Speak Up, Speak Out aims to help you find your voice through spoken word. Travel and accommodations provided for warriors and siblings (ages 13-22) and a chaperone.
See URL in image to sign up today.
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