Rare Classroom: Stevens-Johnson Syndrome
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on
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ALS Drug Facing Possible Rejection by the European Medicines Agency
- June 9, 2023
New Updates are Available on a Phase 2 Trial Evaluating CS1 for PAH
Experimental Therapy for Hemophilia Meets Phase 3 Trial Endpoints
A Newly Initiated Phase 1 Study Will Evaluate AIV007 for Wet AMD, DME
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PATIENT WORTHY SERIES
World Orphan Drug Congress USA 2023: The Forefront of Rare Disease Drug Development
The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023 at the Gaylord National Resort & Convention Center in National Harbor, MD.
Rare Community Profiles: How the Seena Magowitz Foundation is Working to Change the World for People Living with Pancreatic Cancer
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease
Study of the Week: RNA Sequencing Platform Could Improve Pediatric Rare Disease Diagnosis
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we
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Stargardt Disease
OCU410ST Granted Orphan Drug Designation for ABCA4-Associated Retinopathies
ABCA4-associated retinopathies are a group of conditions that cause progressive macular degeneration and vision loss due to ABCA4 mutations. Stargardt disease, cone-rod dystrophy type 3
Metastatic Breast Cancer
A Letter to My Daughter Violet
Written by Libby Wilson- Mommy, Living with Stage IV Breast Cancer Dear Violet – Before I met you, I never loved someone with every morsel
Diabetic retinopathy
Using Teleretinal Imaging Could Improve Diabetic Retinopathy Screening
Our country has an issue when it comes to screening for diabetic retinopathy. In addition to clinics lacking the necessary imaging programs, the compliance
Eastern Equine Encephalitis
Two Cases of Eastern Equine Encephalitis (EEE) Identified in Polk County, FL
Eastern equine encephalitis (EEE) is a rare condition that can be spread through the bites of infected mosquitos. In a majority of cases, birds are
Colorectal cancer
Why are KRAS-Mutated Colorectal Cancer Tumors More Difficult to Treat?
Have you ever thought about your genes and what they do for your body? For example, the KRAS gene—do you know what it does? Normally,
Hemophilia
Rare Community Profiles: From the 1960s to Now: How Two Hemophilia Advocates Have Seen Treatment and Research Progress
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease
Lupus
When Science Gets Personal: A Researcher’s Journey with Lupus
Written by Katherine Tran Lupus is among the most perplexing autoimmune diseases. It can be debilitating when not regulated, with symptoms ranging from butterfly-shaped rashes
Cystinosis
Patients Rising, Cystinosis Awareness
Written by K.M. I have never given much thought to how I would die. Perhaps because my health and longevity has been laid out for
Nonalcoholic fatty liver disease
Non-Alcoholic Fatty Liver Disease (NAFLD) in Pregnancy has Nearly Tripled in the Past Ten Years
In the last decade, NAFLD in pregnancy has nearly tripled in concert with the worldwide increase in diabetes. Mayo Clinic reports that NAFLD in
Multiple Myeloma
That’s My Word: Spreading Multiple Myeloma Awareness in the Black Community
Marsha Calloway-Campbell lives a full life as a lawyer, business owner, wife to her husband Armaray, mother to her three adult daughters, and “Naun” to
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