Rare Community Profiles: Volv’s Innovative Approach Harnesses the Power of AI for Rare Disease Detection
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Rare Community Profiles: Volv’s Innovative Approach Harnesses the Power of AI for Rare Disease Detection

  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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Rare Community Profiles: Astellas Pharma’s Dr. Anthony Yanni Discusses the Success of the First-Ever Patient Advocacy Organization Action Week (PAOAW)
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Rare Community Profiles: Astellas Pharma’s Dr. Anthony Yanni Discusses the Success of the First-Ever Patient Advocacy Organization Action Week (PAOAW)

  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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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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New FDA Regulation on Laboratory Developed Tests Draws Objections from Healthcare Groups
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New FDA Regulation on Laboratory Developed Tests Draws Objections from Healthcare Groups

As reported in Biopharma Dive, the FDA’s decision to strengthen its authority involving laboratory developed tests (LDTs) has drawn considerable objections from various healthcare groups. However, the Agency has held…

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Scientists have Discovered a Connection Between Metabolic Problems in the Brain and Neurological Disorders
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Scientists have Discovered a Connection Between Metabolic Problems in the Brain and Neurological Disorders

  These efforts led to collaboration by the International Brain pH Project that involves 131 scientists in 105 labs from seven countries. The scientists have identified changes in lactate levels…

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Science Simplified: Differences Between a Gene Therapy Clinical Trial and a Cure
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Science Simplified: Differences Between a Gene Therapy Clinical Trial and a Cure

Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…

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Doctors Transplant Genetically Edited Pig Kidney Into 62-Year-Old Man with End-Stage Kidney Disease
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Doctors Transplant Genetically Edited Pig Kidney Into 62-Year-Old Man with End-Stage Kidney Disease

Over 100,000 individuals living in the U.S. are on a waiting list for an organ transplant. An average of 17 people die each day while waiting for a donor organ.…

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Highlights of ACMG 2024
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Highlights of ACMG 2024

Perhaps 70- 85% of rare diseases have a root in genetics, - which is why continuous research and reporting is so critical for those with rare conditions. This is exactly…

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Study Lists Neurological Disease as the Top Cause of Disability and Illness Around the World
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Study Lists Neurological Disease as the Top Cause of Disability and Illness Around the World

Neurological conditions now have the highest rates of disability and ill health worldwide. The Disparity Although more than 80% of the neurological deaths and nervous system disorders occur in lower…

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Rare Community Profiles: Yellow for Yiannis: Angela’s Mission to Advance Research into IRF2BPL
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Rare Community Profiles: Yellow for Yiannis: Angela’s Mission to Advance Research into IRF2BPL

  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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Rare Community Profiles: A Potential Treatment Turning Point for ALK-Positive NSCLC: Dr. Ken Culver Shares Insights from the Phase 3 ALINA Study on Alecensa
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Rare Community Profiles: A Potential Treatment Turning Point for ALK-Positive NSCLC: Dr. Ken Culver Shares Insights from the Phase 3 ALINA Study on Alecensa

  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.…

Continue Reading Rare Community Profiles: A Potential Treatment Turning Point for ALK-Positive NSCLC: Dr. Ken Culver Shares Insights from the Phase 3 ALINA Study on Alecensa
American College of Medical Genetics and Genomics 2024: Advances Promise a Better Future for Rare Disease Patients
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American College of Medical Genetics and Genomics 2024: Advances Promise a Better Future for Rare Disease Patients

The overwhelming majority of rare diseases and conditions have a genetic basis. This means that the disease is the result of a genetic abnormality such as a mutation. These abnormalities…

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