Rare Disease
“Two Rare Diagnoses, One Common Lesson: Start with Genetic Testing”
Every day, parents just like me sit in doctors’ offices searching for answers. They know something isn’t quite right. Maybe their child isn’t meeting milestones.
Some Dialysis Humor, Part 2
AtaiBeckley Completes Patient Dosing in Phase 2b Trial of VLS-01 for Treatment-Resistant Depression
Natalie’s GACI Story
LISTEN TO OUR PODCAST
THE LATEST FROM PATIENT WORTHY
Mesothelioma
New Experimental Drug Offers Hope for Mesothelioma Patients
As reported on Vermont Biz, researchers at the University of Vermont (UVM) and biotechnology company RS Oncology have reported encouraging results from an early-stage clinical
Alzheimer's disease
2026 Alzheimer’s Update
For more than a century Alzheimer’s disease has been thought to be incurable. But a recent study published in the New England Journal of Medicine
Chronic Kidney Disease
Some Dialysis Humor, Part 1
Editor’s Note: This is part 1 of a 3-part caregiver story, submitted to us by Joan Foster, who was a caregiver for her husband Charles’
Hepatocellular Carcinoma
FDA Issues Complete Response Letter for Rivoceranib–Camrelizumab Combination in Advanced Liver Cancer
As reported by The Manila Times, the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to Elevar Therapeutics regarding its
Hemophilia A
ISTH 2026: Long-Term Phase 3 Data Support Denecimig as a Flexible Prophylactic Option for Hemophilia A
As covered by Fairfax Times, Novo Nordisk has reported encouraging long-term findings for its investigational hemophilia A therapy denecimig (Mim8), with interim results from the
Rare Disease
Heal Canada’s Summer 2026 Issue of E3 Advocacy Magazine
Our friends at Heal Canada have launched the newest edition of their magazine, E3 Advocacy! Their digital publication focuses on the importance of patient advocacy,
Rare Disease
FDA Grants Breakthrough Device Designation to Aurenar’s V-Link System for Preventing Cerebral Vasospasm After Brain Hemorrhage
As reported on MobiHealth News, the U.S. Food and Drug Administration (FDA) has awarded Breakthrough Device designation to Aurenar’s V-Link System, a noninvasive neuromodulation technology
Congenital Adrenal Hyperplasia
An Interview with Allyson, a Congenital Adrenal Hyperplasia Patient
Can you take me back to when you first became aware that you had a health condition as a child. Did you overhear the adults
HIV/AIDS
Merck Partners With ACTF to Expand Access to New HIV Treatment Through State ADAP Programs
As reported on PharmaBiz, Merck has announced a new agreement with the ADAP Crisis Task Force (ACTF) aimed at improving access to its recently approved
Hidradenitis suppurativa
GLP-1 Receptor Agonists Associated With Lower Cardiovascular and Inflammatory Risks in Hidradenitis Suppurativa
As reported on MedPage Today, large retrospective study suggests that glucagon-like peptide-1 (GLP-1) receptor agonists may offer broad health benefits for patients with hidradenitis suppurativa
Duchenne Muscular Dystrophy
FDA Sets Early 2027 Review Deadline for Sarepta’s Duchenne Exon-Skipping Therapies Despite Confirmatory Trial Setback
As reported on BioSpace, Sarepta Therapeutics has reached an important regulatory milestone in its effort to secure traditional FDA approval for two Duchenne muscular dystrophy
Pompe Disease
Nexviazyme Achieves Key Phase 3 Milestone in Infants With Pompe Disease, Sanofi Prepares FDA Expansion Filing
As reported on Tech Times, Sanofi has reported positive results from its Phase 3 Baby-COMET study evaluating Nexviazyme (avalglucosidase alfa) in infants with infantile-onset Pompe
Ovarian Cancer
A Doctor-Patient Interview on Ovarian Cancer
Editor’s Note: Patient Worthy is honored to share this interview, featuring Kate, an ovarian cancer survivor, and Dr. Premal Thaker, M.D. “There is fear, and
Rare Disease
FDA Clears Orca Bio’s Tregzi for Adults Undergoing Allogeneic Stem Cell Transplantation for Blood Cancers
As reported on PharmaBiz, the US Food and Drug Administration (FDA) has approved Orca Bio’s Tregzi, a precision-engineered cell therapy designed for use in matched-donor
Sickle Cell Disease
FDA Expands Casgevy Use to Children as Young as 2 Years Old
As reported on FiercePharma, the U.S. Food and Drug Administration (FDA) has approved an expanded indication for Vertex Pharmaceuticals’ gene-editing therapy Casgevy (exagamglogene autotemcel), allowing
Obesity
Ascletis Advances Obesity Pipeline With Two FDA IND Filings for Long-Acting Injectable Candidates
As reported on PharmaBiz, Ascletis Pharma has announced the submission of two Investigational New Drug (IND) applications to the US Food and Drug Administration (FDA)
Spinal Muscular Atrophy
European Commission Expands Approval of Novartis Gene Therapy for Broader SMA Population
As reported on Pharmaceutical Technology, the European Commission has approved Novartis’ gene replacement therapy Itvisma (onasemnogene abeparvovec) for the treatment of children aged two years
FEATURED
UPCOMING EVENTS
SIGN UP FOR OUR NEWSLETTER
Many people don't realize that gastric (stomach) cancer and gastroesophageal junction (GEJ) cancer can develop quietly, with symptoms that may be mistaken for common digestive issues. Persistent heartburn, feeling full after eating small amounts, unexplained weight loss, or difficulty swallowing shouldn't be ignored—especially if they don't improve over time.
While not every symptom means cancer, knowing the potential warning signs and understanding risk factors can help you recognize when it's time to talk with your healthcare provider. Awareness is one of the first steps toward earlier detection and informed care.
We would love to hear from you! Please click the link below to share your story -
ow.ly/4UOp50ZozFp
#GastricCancer #gejcancer #PatientWorthy
For some people living with rare diseases, changes in vision can go beyond blurry eyesight. Seeing flashes or flickering lights can affect daily activities and may be a sign of underlying eye or neurological involvement. Raising awareness of symptoms like these helps highlight the diverse ways rare diseases can affect the body and why changes in vision should never be overlooked.
#rarewordoftheweek #raredisease #rarediseaseawareness #PatientWorthy
Blood Donors are Still Needed for Gigi
