Rare Disease
Heart Attacks and Your Brain
1. A Physical Smoking Gun in the Heart-Brain Connection When people experience cognitive problems, mood drops, or chronic anxiety after surviving a heart attack, the
Phase 3 Trial Highlights Rapid, Sustained Benefit of Veligrotug in Thyroid Eye Disease
- June 21, 2026
FDA Clarifies Marketing Limits for Compounded GLP-1 Drugs Amid Ongoing Enforcement Effort
FDA Clears First Over-the-Counter Continuous Glucose Monitor for Children
FDA Approves Pembrolizumab–Belzutifan Combination for Adjuvant Treatment of High-Risk Clear Cell RCC
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Bronchiectasis
Infex Therapeutics Reports Positive Phase IIa Data for RESP‑X in Bronchiectasis Patients with Pseudomonas Colonisation
As reported on PharmaBiz, Infex Therapeutics has announced encouraging results from a phase IIa clinical study of RESP‑X, an investigational monoclonal antibody being developed for
ADHD
Cingulate Receives FDA Complete Response Letter for ADHD Candidate Over Manufacturing Issues
As reported on FiercePharma, Cingulate Therapeutics has encountered a regulatory setback in its effort to bring its lead attention-deficit/hyperactivity disorder (ADHD) therapy to market, after
MPS III (Sanfilippo Syndrome)
Should vs. Want: Guiding Insights from a Palliative Care Clinician
Editor’s Note: This article was originally written by Joanne Huff, and shared with us by our friends at the Courageous Parents Network. To see the
Alpha-1 antitrypsin deficiency
Efdoralprin Alfa Shows Superior Efficacy Over Standard Augmentation Therapy in AATD Emphysema
As reported on PharmaBiz, new findings from the phase 2 ElevAATe trial suggest that the investigational agent efdoralprin alfa may represent a meaningful advance in
COVID-19
FDA Clears First Oral Medication for Post-Exposure COVID-19 Prevention
As reported on Beckers Hospital Review, the U.S. Food and Drug Administration has authorized ensitrelvir (brand name Xocova) as the first oral antiviral indicated for
Rare Disease
Hidden for Six Weeks: How My Brain Injury Revealed Itself
To this day, I still don’t remember the car accident that changed my life. It was Thursday, August 8, 2024 — a day like any
Limb-girdle muscular dystrophy
FDA Priority Review Signals Potential Breakthrough for Limb-Girdle Muscular Dystrophy
As reported on BioSpace, the U.S. Food and Drug Administration (FDA) has granted priority review to BridgeBio Pharma’s investigational therapy, BBP-418, marking a key regulatory
Ebola
A Deeper Dive into the Ebola Virus
Formally known as Ebola hemorrhagic fever, Ebola is often fatal in humans. The disease is transmitted through contact made with body fluids or blood of
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
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
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Amanda is a lupus advocate, wellness seeker, and believer in the power of resilience. After years of navigating chronic illness, loss, and life's unexpected challenges, she has discovered that strength is often built in the moments we never asked for. In this video, Amanda shares some of the lessons, perspectives, and insights that have helped her navigate life with lupus. Her hope is to remind others that while chronic illness may shape your journey, it does not define your ability to live a happy, meaningful, and fulfilling life. If you are interested in sharing your healthcare journey, click the link: bit.ly/4dV7gru #PatientWorthy #lupus #LN #lupusnephritis #shareyourstory
This Men’s Health Week, we're highlighting the importance of preventive care, regular screenings, and open conversations about health.
Taking charge of your health isn't just about addressing problems when they arise—it's about building habits that support long-term well-being. From physical health to mental health, every step toward better care matters.
#MensHealthWeek #MensHealth #PatientWorthy
Thyroid Eye Disease can change how you see the world and how the world sees you. Because symptoms can develop gradually or be mistaken for something else, many people don’t realize what’s happening right away.
That’s why awareness and testing are so important. A diagnosis is more than a label. It’s a step toward understanding your symptoms, finding the right care, and protecting your vision for the future. If something feels different with your eyes, it deserves attention. The sooner you have answers, the sooner you can take control of your health. Visit patientworthy.com for more information on TED.
#ThyroidEyeDisease #TEDAwareness #EyeHealth #EarlyDetection #PatientWorthy
