Had High BMI as a Child? Your Risk of Chronic Kidney Disease (CKD) in Adulthood May Be Heightened
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Had High BMI as a Child? Your Risk of Chronic Kidney Disease (CKD) in Adulthood May Be Heightened

Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…

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Long-Term Safety and Efficacy Data Available on Vabysmo for Diabetic Macular Edema
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Long-Term Safety and Efficacy Data Available on Vabysmo for Diabetic Macular Edema

Since its development and approval, Vabysmo has been a game-changer in the ophthalmologic space. The injectable VEGF and Ang-2 inhibitor treats diabetic macular edema (DME), macular edema with retinal vein…

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SLEEP 2024: Two Studies Explore Link Between Narcolepsy and Cardiovascular Disease Risk
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SLEEP 2024: Two Studies Explore Link Between Narcolepsy and Cardiovascular Disease Risk

The SLEEP 2024 Annual Meeting took place this year from June 1 to June 5 in Houston, TX. During the course of this premier sleep conference, stakeholders across the sleep…

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STUDY: 33% of Adults with Severe Autoimmune Hemolytic Anemia Responded Well to Add-On IVIG
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STUDY: 33% of Adults with Severe Autoimmune Hemolytic Anemia Responded Well to Add-On IVIG

Prednisone, oral steroids, intravenous hydrocortisone, and even blood transfusions are used to treat autoimmune hemolytic anemia (AIHA). AIHA comprises of warm AIHA, mixed AIHA, or cold agglutinin disease (CAD). Some…

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STUDY: Pitolisant Reduced Fatigue and Daytime Sleepiness in Adults with DM1
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STUDY: Pitolisant Reduced Fatigue and Daytime Sleepiness in Adults with DM1

An estimated 80% or more of people with myotonic dystrophy type 1 (DM1) struggle with severe fatigue and excessive daytime sleepiness (EDS). While most people consider myotonia (inability to contract…

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Researchers Identify Underlying Disease Mechanism in RVCL
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Researchers Identify Underlying Disease Mechanism in RVCL

Researchers have previously linked TREX1 gene mutations to the development of a rare inherited small vessel disease called retinal vasculopathy with cerebral leukoencephalopathy (RVCL). What researchers didn't know was why these genetic…

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Volunteers Needed for Alpers Syndrome Study
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Volunteers Needed for Alpers Syndrome Study

Stemnovate is a biotechnology company working to transform the drug discovery landscape while improving diagnostics. Currently, the company hopes to develop a research platform for rare pediatric mitochondrial diseases such…

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Mirum Pharmaceuticals: Results Available from PBC and PSC Studies on Volixibat
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Mirum Pharmaceuticals: Results Available from PBC and PSC Studies on Volixibat

Mirum Pharmaceuticals has long been a leader in the rare liver disease space, driving forward with a mission of empowering patients and families, and identifying novel therapeutic interventions to help…

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Healthy Skin Transplants Could Successfully Treat Epidermolytic Ichthyosis and Ichthyosis with Confetti
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Healthy Skin Transplants Could Successfully Treat Epidermolytic Ichthyosis and Ichthyosis with Confetti

You've heard of kidney transplants. You've heard of liver transplants. And you've probably even heard of lung transplants. But have you heard of skin transplants? In many cases, skin transplants…

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Rare Community Profiles: White Paper Discusses Access Barriers in IgG4-Related Disease (IgG4-RD)
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Rare Community Profiles: White Paper Discusses Access Barriers in IgG4-Related Disease (IgG4-RD)

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

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First Patient Enrolled in VE303 Study for Recurrent C. Diff
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First Patient Enrolled in VE303 Study for Recurrent C. Diff

An estimated 25% of people with Clostridioides difficile (C. diff) infections experience recurrence following successful antibiotic intervention. After three or more recurrent infections, the risk of developing another infection sits…

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UC Davis Health Researchers Identify Biomarkers for Schizophrenia in 22q11.2 Deletion Syndrome

A 2008 report published in Current Psychiatry Reports found that approximately 1% of people with schizophrenia also have a rare genetic disorder called 22q11.2 deletion syndrome. This suggests that schizophrenia…

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Phase 3 Results: Imsidolimab Provides Positive Outlook in Generalized Pustular Psoriasis (GPP)
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Phase 3 Results: Imsidolimab Provides Positive Outlook in Generalized Pustular Psoriasis (GPP)

Just about one month ago, clinical-stage biotechnology company AnaptysBio announced the availability of positive top-line results from two Phase 3 clinical studies: GEMINI-1 and GEMINI-2. Both studies sought to assess…

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Women More Likely to Develop De Novo Conditions Within One Year of Cushing Syndrome Remission
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Women More Likely to Develop De Novo Conditions Within One Year of Cushing Syndrome Remission

Researchers recently linked Cushing syndrome remission to the development of de novo (first occurrence) diseases, including autoimmune diseases, in various patients. But how did they reach this point? The first…

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ASO Therapy Improves Cellular Function in Timothy Syndrome Brain Organoids
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ASO Therapy Improves Cellular Function in Timothy Syndrome Brain Organoids

Have you heard of antisense oligonucleotides (ASOs)? These short, synthetic strands of nucleic acids are designed to bind specifically to the mRNA transcripts of genes, blocking the production of disease-causing…

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CMT Research Foundation Invests in Study Evaluating Potential Therapy for CMTX1
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CMT Research Foundation Invests in Study Evaluating Potential Therapy for CMTX1

Charcot-Marie-Tooth disease (CMT), though rare, is one of the most commonly inherited neurological disorders and affects peripheral nerves outside of the brain and spinal cord. Multiple CMT subtypes exist based…

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