Rare Disease
Novel Therapies and the FDA
Twenty-six Novel Therapies have been FDA-approved so far this year. Medical journals, including “The Lancet”, see this as a difficult task. The Agency must maintain
A Routine Colonoscopy at 44 Revealed Cancer – Now She’s Raising Awareness
- September 27, 2026
Boundaries, Securities, and Safety: The Unexpected Challenges of Kevin’s PKU Advocacy
Lung Health Starts Early and Needs Protecting Throughout Life
Organoid Advances Could Open New Paths for Spinal Cord and Peripheral Nerve Repair
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Diabetes Type 2
FDA Approves Lilly’s Once-Weekly Onswik Insulin for Adults With Type 2 Diabetes
As reported on PR Newswire, the U.S. Food and Drug Administration has approved Eli Lilly and Company’s Onswik (insulin efsitora alfa-gobe), introducing a once-weekly basal
Rare Disease
The Invisible Kingdom: Reimagining Chronic Illness
Chronic diseases perplex doctors, while leaving patients mysteriously ill Meghan O’Rourke, the author of the best-selling book Invisible Kingdom, recently released an excerpt from this
non-small cell lung cancer
Investigational Immunotherapy Improved Survival in Advanced Squamous NSCLC
As reported on MedPage Today, an experimental immunotherapy called gotistobart helped patients with advanced squamous non-small cell lung cancer (NSCLC) live longer than standard chemotherapy
Gaucher Disease
Sharing Our Stories Is a Gift, and It Costs Us Every Time
Editor’s Note: Patient Worthy is honored to share this essay from Carrie Ostrea. I have been fortunate to share my rare disease daughter’s story for
COVID-19
Nearly Seven Years Later: Why Are So Many Long COVID Patients Still Going Untreated?
A Q&A with Robert Groysman, MD, on why standard tests can miss long COVID—and how individualized care may help patients break the cycle When the
Cancer
Liquid Biopsy and Cancer
The Wall Street Journal opinion editors recently published an article a liquid biopsy test called Galleri, prior to an upcoming FDA review. Key Trial Results
Crohn's disease
The Willem Family’s Medical Challenges
For most of my life, I knew something was wrong with my body. I just never had a name for it. When I was around
Limb-girdle muscular dystrophy
The Speak Foundation Lobbies in Washington: BridgeBio’s Development Hopeful
“For people living with a progressive rare disease, time is measured differently. Research and clinical trials can take years while patients continue to lose strength
Chronic Myelogenous Leukemia
Fourteen Years Living With CML
For World CML Day (September 22nd), Nancie Steinberg reflects on living with chronic myeloid leukemia and how a cancer diagnosis ultimately led her from patient
Aplastic anemia
First Responder Stunned by Severe Aplastic Anemia Diagnosis: Greg’s Story
Editor’s Note: Patient Worthy is honored to share this article from our friends at the Aplastic Anemia and MDS International Foundation. To see the article
MPS III (Sanfilippo Syndrome)
FDA Approves Fayuvi, the First Gene Therapy for Children with Sanfilippo Syndrome Type A
One-time treatment provides a new option for pediatric patients with a rare, progressive genetic disorder As announced in a recent press release, the U.S. Food
Cervical Dystonia
Creating Inner Space to Reduce Pain and Burnout
I want to share this to give some ideas I have found that help me move through difficult days with more steadiness and self compassion,
Bladder cancer
ASTRO Issues First-Ever Radiation Therapy Guideline for Bladder Cancer
As reported on Radiology Business, the American Society for Radiation Oncology (ASTRO) has released its inaugural clinical practice guideline dedicated to the use of radiation
Chronic Kidney Disease
FDA Expands Kerendia Approval to Adults With CKD Linked to Type 1 Diabetes
As reported on Healio, the U.S. Food and Drug Administration has approved finerenone (Kerendia; Bayer) for adults with chronic kidney disease (CKD) associated with type
Rare Disease
Biomarkers
To start, a few words about the immune system: The immune system is comprised of cells, molecules, and biochemicals, and works to maintain human health.
Rare Disease
Planning Accessible Travel: What Actually Makes a Trip Work
Editor’s Note: Patient Worthy is honored to share this article, originally written by Linda Chase of Able Hire. Accessible travel planning means building a trip
Rare Disease
A Guide to Staying Well and Strong for New Caregivers
For new caregivers supporting a parent, partner, or relative at home, the days can turn into an unbroken string of decisions, vigilance, and worry. The
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🤩September is Patient Appreciation Month🤩 At Patient Worthy, we are celebrating the patients who have bravely shared their diagnosis and treatment journey with us. Your stories not only raise awareness, they also help others feel seen, heard, and less alone.
Thank you for turning your experiences into sources of inspiration and hope. Your voice matters, and your stories are making a difference!
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For people navigating complex or rare conditions, there may be more than one health issue happening at the same time. Overlapping symptoms, treatments, and diagnoses can make it difficult to understand what’s connected and what isn’t. Recognizing the full picture is an important part of understanding each patient’s unique health experience and the complexity of managing multiple concerns at once.
#RareWordOfTheWeek #RareDisease #RareDiseaseAwareness #PatientWorthy
The future for recovery and repair of PNIs (peripheral nerve injuries) and SCIs (spinal cord injuries) is looking brighter than ever, as advances in stem cell research continue to open promising new avenues. Read more here, or click the link in our bio!
patientworthy.com/2026/09/25/organoid-advances-could-open-new-paths-for-spinal-cord-and-periphera...
