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
Ultrasounds and Pain
A Systemic Mastocytosis Journey: From Diagnosis to Advocacy
Daraxonrasib Is Promising. But Here’s What Pancreatic Cancer Patients Need to Know First
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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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The world often believes what it can see. But invisible illness is navigating symptoms, limits, appointments, fatigue, and uncertainty that rarely make it to the surface. To those carrying unseen battles every day: you are not alone.
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Myasthenia gravis is a chronic autoimmune neuromuscular condition that affects communication between nerves and muscles, often causing muscle weakness that can fluctuate throughout the day.
Awareness helps foster understanding, encourages earlier recognition of symptoms, supports research efforts, and reminds those living with MG that they are not alone in their journey.
This month we honor the strength, resilience, and determination of the MG community while continuing to advocate for education, support, and progress.
#myastheniagravis #autoimmunedisease #PatientWorthy
We're honored and thrilled to bring you this article by Dr. Avishek Kumar, discussing breaking news on how pancreatic cancer treatment has potentially been changed forever. Read here:
bit.ly/4x5u2pX
#PatientWorthy #Cancer #pancreaticcancer #shareyourstory
