Phenylketonuria
Boundaries, Securities, and Safety: The Unexpected Challenges of Kevin’s PKU Advocacy
15 years ago I released my first PKU project. I never imagined the day would come when I would need to assert my boundaries this
Synapticure: A Nationwide Neurology Network Developed by Utilizing Telehealth
FDA Expands Welireg Use in Kidney Cancer With Lenvima Combination
A Routine Colonoscopy at 44 Revealed Cancer – Now She’s Raising Awareness
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Rare Disease
Lung Health Starts Early and Needs Protecting Throughout Life
World Lung Day 2026 puts a spotlight on lung health across the life course Lung health begins early in life and needs to remain a
Peripheral Nerve Injury
Organoid Advances Could Open New Paths for Spinal Cord and Peripheral Nerve Repair
As reported on News Medical, researchers are increasingly exploring laboratory-grown organoids as potential tools for repairing damage caused by spinal cord and peripheral nerve injuries.
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
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
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Alagille Syndrome Alliance #scholarshipA new opportunity to support your education is coming soon! Applications for the 2026 Alaina K. Hahn Celebratory Scholarship open on October 1st.
This scholarship is open to eligible people with Alagille Syndrome, as well as their siblings, children, and parents or guardians, who are entering or enrolled in an accredited U.S. college, university, or vocational school.
The Alaina Kaitlyn Hahn Celebratory Scholarship supports students pursuing human biological, medical, health science, or allied fields, including social work, mental health, physical or occupational therapy, creative therapy, and animal-assisted therapy.
Learn more about eligibility and how to apply: zurl.co/lxYBl
#AlagilleSyndrome #ALGSA #Scholarships
International Rett Syndrome FoundationMissed our RettEd Webinar Same Gene, Different Rett: Diagnosis, Spectrum, and Family Questions?
The recording is now available to watch on demand. Amitha Ananth and Dominique Pichard break down what diagnostic and classification systems actually measure, where they fit into your child's care plan, and what it looks like to hold the data in one hand and your child in the other.
Watch now: youtu.be/aenao1dSKC0
