Rare Community Profiles: Inozyme’s Catherine Nester Discusses Newborn Screening and the GACI Diagnostic Delay
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Rare Community Profiles: Inozyme’s Catherine Nester Discusses Newborn Screening and the GACI Diagnostic Delay

Rare Community Profiles     Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…

Continue Reading Rare Community Profiles: Inozyme’s Catherine Nester Discusses Newborn Screening and the GACI Diagnostic Delay
A Colon Cancer Diagnosis During Pregnancy Led This Mother to Learn She Had Li-Fraumeni Syndrome
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A Colon Cancer Diagnosis During Pregnancy Led This Mother to Learn She Had Li-Fraumeni Syndrome

For the first two months of her pregnancy, Kristine Koser felt pretty good. Koser and her husband, Andrew, couldn't wait to welcome their daughter Aubrey into the world. Everything seemed…

Continue Reading A Colon Cancer Diagnosis During Pregnancy Led This Mother to Learn She Had Li-Fraumeni Syndrome

After a 26-year Journey, I Created My Own Diet and Healed My Eosinophilic Esophagitis (EoE)

Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…

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Study: Risk Factors for Paroxysmal Nocturnal Hemoglobinuria Thromboembolic Events
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Study: Risk Factors for Paroxysmal Nocturnal Hemoglobinuria Thromboembolic Events

Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…

Continue Reading Study: Risk Factors for Paroxysmal Nocturnal Hemoglobinuria Thromboembolic Events
FACIT-Fatigue Scale: A 5-Point Change is Important for Paroxysmal Nocturnal Hemoglobinuria
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FACIT-Fatigue Scale: A 5-Point Change is Important for Paroxysmal Nocturnal Hemoglobinuria

Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…

Continue Reading FACIT-Fatigue Scale: A 5-Point Change is Important for Paroxysmal Nocturnal Hemoglobinuria

Ryan’s Life Changed from being a Fire Fighter to Being Diagnosed with Creutzfeldt-Jakob Disease

Dozens of Ryan Wilson Palmer’s family and friends recently celebrated an early birthday after he received a devastating diagnosis of Creutzfeldt-Jakob disease. A few months ago 49-year-old Ryan Palmer of…

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Rare Community Profiles: Strong Like a Mother: How Judy Has Remained Steadfast Through Her IgAN Journey
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Rare Community Profiles: Strong Like a Mother: How Judy Has Remained Steadfast Through Her IgAN Journey

Rare Community Profiles     Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…

Continue Reading Rare Community Profiles: Strong Like a Mother: How Judy Has Remained Steadfast Through Her IgAN Journey

Beam Therapeutics Announces a First in Human Testing of Gene Editing Medicine for Cancer  

Beam Therapeutics (Beam) has developed a technique called base editing. The treatment is called BEAM 201. The first patient has been dosed according to a recent statement by the company…

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September 24 is FH Awareness Day!
Xanthoma is an easy sign of high cholesterol conditions like HeFH. If you see this, please go to the doctor immediately! Source: Wikipedia

September 24 is FH Awareness Day!

An estimated 1.3 million people across the United States, and 30 million people globally, live with familial hypercholesterolemia (FH). This rare inherited form of high low-density lipoprotein (LDL) cholesterol remains…

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Researchers Track Gene Therapy’s Impact on Neural Connections in Hurler Syndrome
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Researchers Track Gene Therapy’s Impact on Neural Connections in Hurler Syndrome

617 words (source - 3% match) vs. 452 words (mine - 4% match) As our healthcare field continues to innovate and grow, we have seen more conversations regarding gene therapy…

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Rare Classroom: Arthrogryposis Multiplex Congenita
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Rare Classroom: Arthrogryposis Multiplex Congenita

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 some of the rarest, most…

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