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’
“Two Rare Diagnoses, One Common Lesson: Start with Genetic Testing”
New Experimental Drug Offers Hope for Mesothelioma Patients
2026 Alzheimer’s Update
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THE LATEST FROM PATIENT WORTHY
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
Amyloidosis
BridgeBio Highlights Potential Kidney Benefits of Attruby in ATTR-CM
As reported on BioSpace, BridgeBio Pharma has reported new findings suggesting that its transthyretin (TTR) stabilizer, Attruby (acoramidis), may help preserve kidney function in patients
Hidradenitis suppurativa
Sonelokimab Shows Sustained One-Year Benefits in Hidradenitis Suppurativa, With Encouraging Early Results in Adolescents
As reported on Healio, new phase 3 data suggest that sonelokimab may offer durable disease control for patients with moderate to severe hidradenitis suppurativa (HS),
non-small cell lung cancer
Good News for Lung Cancer Patients
New research from the 2026 American Society of Clinical Oncology (ASCO) annual meeting, and presented on Cure Today, highlights an encouraging new treatment on the
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@BHDfoundation # Birt-Hogg-Dubé syndrome #researchThere’s still time to participate in this important research!
Researchers are collecting facial photographs (selfies) to help improve earlier detection of Birt-Hogg-Dubé Syndrome and understand how it changes over time.
You may be eligible if you are:
• 18 or older
• Diagnosed with BHD
• Living in the US
If you would like to take part, please use the QR code on the flyer or sign up here:
redcap.stanford.edu/surveys/?s=KM4XEYA8HEN7LWJ3
Dr. Alphie Yip at Stanford University has confirmed they have recieved a very encouraging response so far, but more participants are still needed.
Please consider participating or sharing to help support this promising research. Thank you.
OPMD Association Non-Profit for Oculopharyngeal Muscular DystrophyCheck out our latest Summer 2026 Newsletter and sign up for future editions, opmd.org/newsletter/
Neurofibromatosis Network #painResearchers at Cincinnati Children's have identified a potential new way to relieve chronic pain linked to NF1. The new findings suggest that pain in NF1 may begin before tumors appear and may be driven by abnormal signaling from Schwann cells, which normally support and protect nerves. Read more: www.nfnetwork.org/pages-news/researchers-uncover-possible-cause-of-pain-in-neurofibromatosis-type...
#neurofibromatosis #NF #NF1
