Rare Community Profiles: How Two Parents Are Working to Change the Narrative Around CASK Gene Disorder
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Rare Community Profiles: How Two Parents Are Working to Change the Narrative Around CASK Gene Disorder

  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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May is Lupus Awareness Month!
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May is Lupus Awareness Month!

Also known as systemic lupus erythematosus (SLE), lupus is a chronic autoimmune disorder in which the immune system mistakenly attacks different parts of the body such as the joints, kidneys,…

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Rare Community Profiles: When Sierra’s Son Was Diagnosed with Warsaw-Breakage Syndrome (WABS), She Knew She Needed to Make a Difference
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Rare Community Profiles: When Sierra’s Son Was Diagnosed with Warsaw-Breakage Syndrome (WABS), She Knew She Needed to Make a Difference

  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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April 30th is the First-Ever AMKD Awareness Day. Here’s What You Need to Know
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April 30th is the First-Ever AMKD Awareness Day. Here’s What You Need to Know

  On April 30, 2024, the American Kidney Fund (AKF) will be organizing the first annual APOL1-mediated kidney disease (AMKD) Awareness Day. This awareness day will be a time to…

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Rare Community Profiles: How a Clinical Study Changed Dana’s Trajectory After Desmoid Tumor Recurrence
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Rare Community Profiles: How a Clinical Study Changed Dana’s Trajectory After Desmoid Tumor Recurrence

  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: How Kenzi is Navigating the Challenges of MCTD, FCAS, and Medical PTSD
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Rare Community Profiles: How Kenzi is Navigating the Challenges of MCTD, FCAS, and Medical PTSD

  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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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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Rare Community Profiles: Revving for Hope: Raising HoFH Awareness in One Ride
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Rare Community Profiles: Revving for Hope: Raising HoFH Awareness in One Ride

  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: How Beth and Madison Advocated for Public Funding for the First Cystic Fibrosis (CF) Modulator in Canada
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Rare Community Profiles: How Beth and Madison Advocated for Public Funding for the First Cystic Fibrosis (CF) Modulator in Canada

  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: The Urgent Need for a Cure for Niemann-Pick Type C: Two Parents Share Their Perspectives
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Rare Community Profiles: The Urgent Need for a Cure for Niemann-Pick Type C: Two Parents Share Their Perspectives

  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: Kristi’s Fight to Combat Misconceptions about Prader-Willi Syndrome (PWS): “Our Children Have PWS; They Are Not PWS”
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Rare Community Profiles: Kristi’s Fight to Combat Misconceptions about Prader-Willi Syndrome (PWS): “Our Children Have PWS; They Are Not PWS”

  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 Mother-Son Pair Discuss the Importance of Wellbeing in Managing NF2-Related Schwannomatosis (NF2)
Jo and Oscar. Photo courtesy of Jo Ward

Rare Community Profiles: A Mother-Son Pair Discuss the Importance of Wellbeing in Managing NF2-Related Schwannomatosis (NF2)

  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: 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 Mother’s Journey to Raise 4H Leukodystrophy POLR3B Awareness and Change Her Son’s Life
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Rare Community Profiles: A Mother’s Journey to Raise 4H Leukodystrophy POLR3B Awareness and Change Her Son’s Life

  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: Could It Be HCM? A Mother-Daughter Duo Raise Hypertrophic Cardiomyopathy Awareness
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Rare Community Profiles: Could It Be HCM? A Mother-Daughter Duo Raise Hypertrophic Cardiomyopathy Awareness

  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.…

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Rare Community Profiles: For Rare Disease Week, Barth Syndrome Advocates Took to Capitol Hill to Urge the FDA to Review NDA for Elamipretide

  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: For Rare Disease Week, Barth Syndrome Advocates Took to Capitol Hill to Urge the FDA to Review NDA for Elamipretide