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
FDA Grants Breakthrough Therapy Designation to Pemafibrate for Primary Biliary Cholangitis
- August 5, 2026
Enfortumab Vedotin/Pembrolizumab Regimen Outperforms Neoadjuvant Chemotherapy in Resectable MIBC
Ebola Outbreak and Lessons Learned
How to Improve the Chances of Getting Useful MCAS Test Results
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THE LATEST FROM PATIENT WORTHY
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
Breast Cancer
Refuse to Disappear
Editor’s Note: Patient Worthy is honored to share this article from our friend Abigail Johnston, originally published on her blog No Half Measures. To see
C3 Glomerulopathy
FDA Approved Therapy for Rare and Severe Kidney Disease for Children Twelve or Older
In recent reports on MSN and AdventLS, it was shared that, as a result of the pioneering efforts of Dr. Carla Nester and researchers at
Rare Disease
Authentic Data is Becoming an Asset Class
Our friends and contributors at the TREND Community have provided us with another enlightening article, this time about high-quality human-generated data becoming a scarce and
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Before a diagnosis, a good day might have felt ordinary. After a diagnosis, it can become something you truly appreciate.
Living with a rare disease or chronic illness often changes your perspective. The days with fewer symptoms, more energy, or the chance to simply feel like yourself can become the days you stop taking for granted.
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Annual Meeting and Patient Learning Academy
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The ALD Connect Annual Meeting and Patient Learning Academy brings together the ALD community to connect, learn, and advance ALD research.
