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  • News
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  • CME/CE
  • Phase 1/2 Trial to Assess CRISPR Therapeutics’ CAR T Therapy in NMOSD and Related Diseases
    CRISPR-Cas9 concept art
  • Ifezuntirgene Inilparvovec, uniQure’s HD Gene Therapy, Slows Functional Decline Through Month 48
    Clinical trial
  • Hermansky-Pudlak Syndrome: A Multisystem Disease That Often Leads to Pulmonary Fibrosis
    Kristen Brantner with Cassandra Mendez and Kylee Brantner.
  • General Medicine

    For Cancer Care, the AI Revolution Has Already Begun, Dr. Douglas Flora Says
    Copies of "Rebooting Cancer Care"
  • Rare Cancer Day Shines a Light on Diseases That Together Make Up 25% of All Cancers

Latest News

More News
  • Donidalorsen Approved in Canada for HAE Prevention
  • Unexplained Neurologic Symptoms May Signal Wilson Disease
  • Conduction Disease Precedes Hypertrophy in Men Who Carry Late-Onset Fabry Disease Variant
  • Caregivers of People With Dravet Syndrome, Other DEEs Want Their Providers to Talk About SUDEP
  • High Clinical Heterogeneity Seen in Chinese Patients With ATTR
  • Quantitative CT Could Predict Disease Progression in PPF
  • HD Mouse Model Shows Sex-Dependent Mild Cardiac Dysfunction, Neurometabolic Changes
  • Serum Extracellular Vesicles Distinguish CLL and Predict Treatment Need
  • Case Report: Polycythemia Vera May Drive Secondary FSGS
  • CORE-5 Social Risk Screening Tool Reveals Unmet Needs in Black Patients With Generalized MG
  • FDA to Consider Fenebrutinib, BTK Inhibitor for Relapsing and Primary Progressive MS
  • ANCA Positivity Not an Independent Predictor of ESKD in GPA or MPA

Latest Features

More Features
  • Kristen Brantner with Cassandra Mendez and Kylee Brantner.
    Hermansky-Pudlak Syndrome: A Multisystem Disease That Often Leads to Pulmonary Fibrosis
    By Larry Luxner
    HPS has 11 subtypes; 100% of patients with type 1, 2, or 4 will develop pulmonary fibrosis, usually by their mid-30s.
  • Copies of "Rebooting Cancer Care"
    For Cancer Care, the AI Revolution Has Already Begun, Dr. Douglas Flora Says
    By Larry Luxner
    In his book, Dr. Flora explains how artificial intelligence can finally match cancer's relentless ability to evolve and adapt.
  • Rare Cancer Day Shines a Light on Diseases That Together Make Up 25% of All Cancers
    By Larry Luxner
    Rare Cancer Day takes place annually on September 23.
  • Andrew Wilson, MD, at the ATS International Conference
    With Fazirsiran Trial Underway, Takeda Seeks Answers in AATD-Related Liver Disease
    By Larry Luxner
    Experts believe treatments for AATD-related liver disease will be available in 5 to 10 years.
  • Colleen Hall, Kimberly Ku, MD, and Vera Luxner
    Online and in Person, Podcaster Kimberly Ku Promotes ‘Empathetic Communications’
    By Larry Luxner
    Dr. Ku, an oncologist, hosts the podcast, “Val-You Based Care,” that emphasizes empathy and building trust in health care.

Conference Coverage

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conference coverage
Featured Conference
Society of Hematologic Oncology Annual Meeting

Latest Patient Perspectives

More Patient Perspectives
  • close-up of doctor and patient talking
    Creating Space for the Next Question in FSGS Care
    A mother of 2 sons living with FSGS suggests that availability and timing plays a major role in addressing patients' questions.
    By Jaime Albright Henighan, MSW, FSGS Contributor
    October 2, 2026
  • Closeup of two people's feet walking on pavement
    A Patient With Pompe Disease Asks You to Take a Walk in His Shoes
    If health care professionals could experience Pompe disease firsthand, they’d learn more than any textbook or lab result could tell them.
    By Bruce Campbell, Pompe Disease Contributor
    October 2, 2026
  • woman walking up stairs using handrail
    Are You Asking Your Patients With ATTR-CM the Right Questions?
    Patients with ATTR-CM regularly make adaptations to accommodate their progressive disease. Doctors could benefit from knowing about these changes.
    By Faye Adams, ATTR-CM Contributor
    October 1, 2026
  • Physician offering AATD patient education
    How Patient Education Can Strengthen AATD Advocacy
    When patients with AATD have a strong understanding of their rare disease, they can help raise awareness and better advocate for themselves.
    By Alyssa Gloor, AATD Contributor
    September 30, 2026

Latest HCP Insights

More Insights
  • Serum tryptase testing; SM
    The Complex Relationship Between SM and Hereditary Alpha-Tryptasemia
    By Ariana Boswell, MPH
    Hereditary alpha-tryptasemia and SM exhibit overlapping biochemical and clinical features that may present challenges for accurate diagnosis.
  • Liver Health
    Understanding Kidney Involvement in Primary Biliary Cholangitis 
    By Ariana Boswell, MPH
    Emerging research supports a potential association between primary biliary cholangitis (PBC) and kidney disease.
  • Treatment decisions
    Advances in MCL Therapy Make Treatment Sequencing More Critical
    By Megan Garlapow, PhD
    MCL therapy is shifting toward targeted and immune-based approaches, making the order of treatment increasingly consequential.
  • Blood biomarker research
    Immunosenescence in IPF: Biomarkers and Targeted Therapies
    By Jovana Nikolic, MD
    Immunosenescence offers a novel framework for IPF pathogenesis and treatment, though associated biomarkers and therapies await clinical validation.
  • Coronavirus illustration
    How Viral Infections Influence Myasthenia Gravis
    By Ariana Boswell, MPH
    Growing evidence suggests viral infections can exacerbate myasthenia gravis (MG) or even contribute to its development.

Latest Rare Care Podcast

More Episodes
  • An Interview With Dr. Doug Flora on How AI Can Help Diagnose and Treat Cancer
  • An Interview With Donna Appell, Founder of the Hermansky-Pudlak Syndrome Network
  • An Interview With Rich Savino, Grandfather of a Boy With Duchenne Muscular Dystrophy
  • An Interview With Oncologist Dr. Kimberly Ku, MD, on ‘Empathetic Communications’
  • An Interview With Christine Clemson, PhD, of Entrada Therapeutics
  • An Interview With Avril Daly, President of Eurordis-Rare Diseases Europe

Quizzes

More Quizzes
  • Test Your Knowledge About PPF Life Expectancy
  • Test Your Knowledge About the Epidemiology of FCS
  • Test Your Knowledge About the Pathophysiology of PBC
  • Test Your Knowledge About the Etiology of MCL
  • Test Your Knowledge About the Complications of MCL
  • Test Your Knowledge About PPF Complications
  • Test Your Knowledge About the Prognosis of FSGS
  • Test Your Knowledge About PPF Guidelines
  • Test Your Knowledge About the Comorbidities of FSGS
  • Test Your Knowledge About Therapies for PBC
  • Test Your Knowledge About the Diagnosis of CHAPLE Disease
  • Test Your Knowledge About the Clinical Features of TK2d

Rare Diseases

More Diseases
  • Achondroplasia
  • Acromegaly
  • Acute Lymphoblastic Leukemia
  • Alagille Syndrome
  • Alpha-1 Antitrypsin Deficiency
  • ANCA-Associated Vasculitis
  • Cholangiocarcinoma
  • Chronic Inflammatory Demyelinating Polyneuropathy
  • Chronic Lymphocytic Leukemia
  • Cold Agglutinin Disease
  • Cystic Fibrosis
  • Diffuse Large B-Cell Lymphoma
  • Dravet Syndrome
  • Duchenne Muscular Dystrophy
  • Fabry Disease
  • Familial Chylomicronemia Syndrome
  • Fetal and Neonatal Alloimmune Thrombocytopenia
  • Focal Segmental Glomerulosclerosis
  • Friedreich Ataxia
  • Gastrointestinal Stromal Tumor
  • Gaucher Disease
  • Generalized Pustular Psoriasis
  • Hemolytic Disease of the Fetus and Newborn
  • Hemophilia
  • Hepatocellular Carcinoma
  • Hereditary Angioedema
  • Huntington Disease
  • Idiopathic Pulmonary Fibrosis
  • IgG4-Related Disease
  • Immune Thrombocytopenia
  • Lennox-Gastaut Syndrome
  • Limb-Girdle Muscular Dystrophy
  • Long Chain Fatty Acid Oxidation Disorder
  • Lysosomal Acid Lipase Deficiency
  • Mantle Cell Lymphoma
  • Medullary Thyroid Carcinoma
  • Multiple Sclerosis
  • Myasthenia Gravis
  • Myelodysplastic Syndromes
  • Myelofibrosis
  • Neurofibromatosis Type 1
  • Neuromyelitis Optica Spectrum Disorder
  • Paroxysmal Nocturnal Hemoglobinuria
  • Pompe Disease
  • Prader-Willi Syndrome
  • Primary Biliary Cholangitis
  • Primary Central Nervous System Lymphoma
  • Progressive Pulmonary Fibrosis
  • Pulmonary Arterial Hypertension
  • Sickle Cell Disease
  • Spinal Muscular Atrophy
  • Systemic Mastocytosis
  • Systemic Sclerosis
  • Thymidine Kinase 2 Deficiency
  • Transthyretin Amyloid Polyneuropathy
  • Transthyretin-Mediated Amyloid Cardiomyopathy
  • Warm Autoimmune Hemolytic Anemia
  • Wilson Disease
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