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Rare Disease

Synapticure: A Nationwide Neurology Network Developed by Utilizing Telehealth

Patients in the United States wait an average of 34 to 50 days (roughly 1 to 1.5 months) for an initial neurology appointment, with wait

Renal Cell Carcinoma

FDA Expands Welireg Use in Kidney Cancer With Lenvima Combination

As reported on FiercePharma, Merck & Co.’s Welireg has secured an expanded FDA approval that moves the drug earlier in the treatment of advanced kidney

Colorectal cancer

A Routine Colonoscopy at 44 Revealed Cancer – Now She’s Raising Awareness

I thought I wanted a second baby. My son was an adult, and despite having an adorable Frenchie, I was ready to take steps to

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

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