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  • Better Blood Tests Could Protect More Newborns From Hemolytic Disease
    Kleihauer-Betke test
  • IN013 for Wilson Disease Receives FDA Rare and Orphan Status
    FDA sign
  • A Kansas Man’s Lifelong Struggle With Limb-Girdle Muscular Dystrophy
    Donavon Decker
  • Regional Pockets of HAE Persist Due to Founder Effect, Affecting Generations of Families
    John Anderson
  • New Prader-Willi Therapy Gives Parents Rare Hope for Managing Hyperphagia

Latest News

More News
  • Short-Read IGH Sequencing Achieves Clinical-Grade Accuracy in CLL
  • Achieving Remission at Transplant Time Enhances Survival in Patients With CLL
  • Patients at MGFA Conference Offer Insights on Living With Myasthenia Gravis
  • Central Adrenal Insufficiency Is a Rare, Life-Threatening Manifestation of PWS
  • CMV Reactivation Post-HCT May Prevent Relapse and Improve Survival in ALL
  • FNAIT Linked to Maternal Autoimmune Disease and Psychological Distress
  • Recognizing Rare Platelet Antigens Improves FNAIT Diagnosis
  • Impaired IgG Fucosylation Linked to Severity in FNAIT and HDFN
  • Researchers Identify Possible Biomarkers Associated With Inhibitor Development
  • Fidanacogene Elaparvovec Shows Lasting Benefits for Hemophilia B
  • Achieving Transfusion Independence Improves Survival in Lower-Risk MDS
  • Novel ML-Based MRI Radiomics Model Differentiates PCNSL From GB and BM

Latest Features

More Features
  • Donavon Decker
    A Kansas Man’s Lifelong Struggle With Limb-Girdle Muscular Dystrophy
    By Larry Luxner
    Dutch filmmaker Daimon Xanthopoulos recently produced a 25-minute documentary about Donavon Decker’s life and relationships.
  • John Anderson
    Regional Pockets of HAE Persist Due to Founder Effect, Affecting Generations of Families
    By Larry Luxner
    HAE affects approximately 1 in 50,000 people globally, but is far more prevalent in a few select geographic regions.
  • New Prader-Willi Therapy Gives Parents Rare Hope for Managing Hyperphagia
    By Larry Luxner
    Diazoxide choline is believed to activate the ATP-sensitive potassium channel, which may decrease secretion of peptides that regulate appetite.
  • Lisa Butler
    GBS/CIDP Foundation Helps Promote Awareness of Rare Debilitating Neuropathy
    By Larry Luxner
    The GBS/CIDP Foundation International aims to provide support, with an emphasis on patients and caregivers meeting each other.
  • newborn screening panel
    Advocates Seek Solutions After HHS Ends Newborn Screening Advisory Panel
    By Larry Luxner
    Newborn screening in the US currently identifies about 14,000 babies with serious conditions annually.

Conference Coverage

MORE MEETINGS
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WODC 2025

World Orphan Drug Congress USA

Latest Patient Perspectives

More Patient Perspectives
  • glucocorticoids are first-line therapy for IgG4-RD
    Steroids: The Good, the Bad, the Ugly for ITP
    Patients with a relapse of immune thrombocytopenia (ITP) are often prescribed steroid treatment, which can come with difficult side effects.
    By Kristy Coleman, ITP Contributor
    May 9, 2025
  • Atrial fibrillation
    Is There a Connection Linking AATD and Atrial Fibrillation?
    Knowing a potential connection between alpha-1 antitrypsin deficiency (AATD) and atrial fibrillation helps patients be aware of complications.
    By Alyssa Gloor, AATD Contributor
    May 9, 2025
  • male friends
    Telling Others About CAD Is No Easy Task
    Patients with cold agglutinin disease (CAD) can find it difficult to explain their rare disease diagnosis or living experience to others.
    By Alithea Athans, CAD Contributor
    May 7, 2025
  • Woman speaking at conference
    Finding Inspiration, Deeper Knowledge at WODC 2025
    I am still excited and motivated by what I experienced at this year's World Orphan Drug Congress.
    By Tara Keith, SM Contributor
    May 7, 2025

Latest HCP Insights

More Insights
  • Antenatal pregnancy care
    The Importance of Antenatal Care in Pregnancies at Risk of FNAIT
    By Ryner Lai, MBBS
    Basic, adequate antenatal care is vital in pregnancies at risk of fetal and neonatal alloimmune thrombocytopenia.
  • Women with hemophilia
    Females Can Have Hemophilia Too
    By Ryner Lai, MBBS
    Over the last few years, there has been increased recognition that female hemophilia carriers may suffer from significant bleeding as well.
  • ultrasound testing
    Addressing the Muti-Faceted Burden of Achondroplasia
    By Ryner Lai, MBBS
    Studies demonstrate that the burden of an achondroplasia diagnosis is high, taking a toll on both the patient and their family.
  • Pivoting Toward Patient-Reported Outcomes in Systemic Mastocytosis
    By Ryner Lai, MBBS
    Increasingly, modern medicine is making room for patients to better express themselves through self-reported outcomes, especially for rare diseases like SM.
  • Rituximab May Play a Key Role in Refractory CIDP
    By Ryner Lai, MBBS
    Studies suggest that rituximab may have a significant role to play in patients who have been diagnosed with refractory CIDP.

Latest Rare Care Podcast

More Episodes
  • An Interview With Award-Winning MDA Patient Advocate Donavon Decker
  • An Interview With Hereditary Angioedema Expert Dr. John Anderson
  • An Interview With Chris Lewis, Son of Comedian Jerry Lewis
  • An Interview With Lisa Butler, CEO of the GBS/CIDP Foundation
  • An Interview With Huntington Disease Activist Jimmy Pollard
  • An Interview With MDA 2025 National Ambassador Lily Sander

Quizzes

More Quizzes
  • Test Your Knowledge About Vasculitis
  • Test Your Knowledge About the Symptoms of MF
  • Test Your Knowledge About the Comorbidities of Achondroplasia
  • Test Your Knowledge About the Pathophysiology of GPP
  • Test Your Knowledge About a Study on the Effect of Acoramidis in ATTR-CM
  • Test Your Knowledge About the Etiology of Achondroplasia
  • Test Your Knowledge About the Etiology of PAH
  • Test Your Knowledge About the Clinical Features of IgG4-RD
  • Test Your Knowledge About the Treatment of Achondroplasia
  • Test Your Knowledge About Experimental Therapies for HDFN
  • Test Your Knowledge About the Diagnosis of Achondroplasia
  • Test Your Knowledge About the Genetics of Chronic Lymphocytic Leukemia

Rare Diseases

More Diseases
  • Achondroplasia
  • 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
  • Fetal and Neonatal Alloimmune Thrombocytopenia
  • 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
  • Long Chain Fatty Acid Oxidation Disorder
  • Lysosomal Acid Lipase Deficiency
  • Medullary Thyroid Carcinoma
  • Multiple Sclerosis
  • Myasthenia Gravis
  • Myelodysplastic Syndromes
  • Myelofibrosis
  • Neuromyelitis Optica Spectrum Disorder
  • Paroxysmal Nocturnal Hemoglobinuria
  • Pompe Disease
  • Prader-Willi Syndrome
  • Primary Biliary Cholangitis
  • Primary Central Nervous System Lymphoma
  • Pulmonary Arterial Hypertension
  • Sickle Cell Disease
  • Spinal Muscular Atrophy
  • Systemic Mastocytosis
  • Systemic Sclerosis
  • Thymidine Kinase 2 Deficiency
  • Transthyretin Amyloid Polyneuropathy
  • Transthyretin-Mediated Amyloid Cardiomyopathy
  • Wilson Disease
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