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  • CME/CE
  • FDA Approves First-of-Its-Kind Monthly Treatment for HAE
    Medication autoinjector
  • Nerve Ultrasound and Magnetic Resonance Neurography Could Be Useful in CIDP Monitoring
    Diagnosis concept
  • Emerging Solutions Could Make TK2d Easier to Recognize and Treat
    mitochondria and DNA illustration
  • SMA Continues to Challenge Patients Who Couldn’t Be Treated Early in Infancy
    Lyza Weisman and Soraya Perales
  • PoNS Neurostimulation Device Helps to Treat Gait Problems, Patients With MS Say

Latest News

More News
  • Uniform Management Strategy Needed in Immune-Mediated IgG4-RD HP
  • IgG4-ROD May Present With Swelling or Enlargement of Ophthalmic Tissues
  • Periostin Is a Potential Biomarker in Orbital IgG4-RD
  • IgG4-Related Kidney Disease With Monoclonal Gammopathy
  • Deep Learning Could Improve Hemodynamic Assessment in Pediatric PAH
  • Thrombocytopenia a Key Early Predictive Marker of Sepsis Among Newborns
  • Teclistamab May Have Therapeutic Potential in MG
  • Interventions to Reduce Maternal Separation Anxiety Can Be Effective
  • Patients With MF Could Benefit From Posttransplant Cyclophosphamide Administration
  • CSF Biomarkers Could Guide SMA Therapy
  • New Model Could Identify Patients With MF Likely to Have Poor Outcomes After Allo-HCT
  • Ropeginterferon Alfa-2b May Not Be Effective in Patients With MF

Latest Features

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  • mitochondria and DNA illustration
    Emerging Solutions Could Make TK2d Easier to Recognize and Treat
    By Kyle Habet, MD
    TK2d has long existed in a therapeutic void, with patients having few, if any, disease-modifying treatment options.
  • Lyza Weisman and Soraya Perales
    SMA Continues to Challenge Patients Who Couldn’t Be Treated Early in Infancy
    By Larry Luxner
    Lyza Weisman spoke at the recent MDA Clinical & Scientific Conference of no longer being able to do the 'little things' that make a life.
  • PoNS Neurostimulation Device Helps to Treat Gait Problems, Patients With MS Say
    By Larry Luxner
    Kerrie Walters first heard of PoNS in a chance encounter with TV personality Montel Williams, who also has MS and difficulties with gait.
  • As Awareness of Huntington Grows Through Help 4 HD and Others, Hope Is Building
    By Larry Luxner
    Lauren Holder, 39, was diagnosed with Huntington disease a year after her father died from it.
  • 15th International Conference on Myasthenia and Related Disorders
    Myasthenia Gravis, a Rare Disease, Is Rarer Still When It Strikes Children
    By Larry Luxner
    Teenagers account for an estimated 10% to 15% of all MG cases in Europe and North America.

Conference Coverage

MORE MEETINGS
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WODC 2025

World Orphan Drug Congress USA

Latest Patient Perspectives

More Patient Perspectives
  • Smartphone app
    Smartphones and AI: Tools to Empower the Fight Against MS
    Phone apps integrated with AI may be able to track, analyze, and predict multiple sclerosis (MS) progression, enabling patient empowerment.
    By Ed Tobias, MS Contributor
    June 30, 2025
  • Ill Asian woman in bed
    Relapse and Setbacks: How to Be Flexible With My ITP
    Learning to live as an individual with immune thrombocytopenia (ITP) means learning to live with relapses and setbacks.
    By Kristy Coleman, ITP Contributor
    June 30, 2025
  • Monitoring pregnancy-HDFN
    Meeting the Emotional and Physical Demands of a High-Risk Pregnancy
    Kell antibodies are notoriously unpredictable compared to other alloantibody types, such as anti-C, anti-Fyª, that are associated with HDFN. 
    By Emily Hamby
    June 27, 2025
  • Uplizna for NMOSD
    Are Stem Cell Transplants the New Hope for Treating NMO?
    As someone living with neuromyelitis optica (NMO), my initial reaction to the words, “stem cell transplant,” is to run in the opposite direction.
    By Jaimie Sheil, CIDP Contributor
    June 27, 2025

Latest HCP Insights

More Insights
  • Labor and birth
    FNAIT Complications and Care Today
    By Ryner Lai, MBBS
    FNAIT complications can be life-threatening; as such, important strategies must be in place to prevent the worse.
  • Patient questionnaire
    The Need for Tailored Tools to Assess QoL in Achondroplasia
    By Juliana Campos, PhD
    A comprehensive review of QoL tools used in achondroplasia reveals the urgent need for more precise, validated measures.
  • Teen with doctor
    Elderly Patients With ATTR-CM May Benefit From Tafamidis Use
    By Ryner Lai, MBBS
    Tafamidis appears to be effective in extending survival even among very elderly patients with transthyretin amyloid cardiomyopathy.
  • Medical knowledge
    Appraising Currently Available Therapeutic Strategies in ATTR-PN
    By Ryner Lai, MBBS
    Researchers appraised the strengths of currently available therapeutic strategies in managing transthyretin amyloid polyneuropathy.
  • Systemic mastocytosis risk/diagnostics
    Advancing SM Diagnosis With a Standardized Protocol
    By Juliana Campos, PhD
    There are gaps in SM diagnostics and researchers believe there would be advantages to implementing a system-wide screening algorithm.

Latest Rare Care Podcast

More Episodes
  • An Interview With Dr. Ashley Stanley-Copeland of Dell Children’s Medical Center in Austin, Texas
  • An Interview With Dr. Sithara Ramdas on Neonatal and Juvenile Myasthenia Gravis
  • An Interview With Lyza Weisman on Living With Spinal Muscular Atrophy
  • An Interview With Marcus Delatte, PhD, on Using Cannabinoids to Treat Rare Seizure Disorders
  • An Interview With Lucy Culp, the Leukemia & Lymphoma Society VP of State Government Affairs
  • An Interview With Dan Lier, a Partner With the Nonprofit Group ‘Somebody To Talk To’

Quizzes

More Quizzes
  • Test Your Knowledge About the Differential Diagnosis of Achondroplasia
  • Test Your Knowledge About the Diagnosis of GPP
  • Test Your Knowledge About the Epidemiology of GPP
  • Test Your Knowledge About the Treatment of CIDP
  • Test Your Knowledge About the Epidemiology of TK2d
  • Test Your Knowledge About the Diagnosis of TK2d
  • Test Your Knowledge About the Complications of IgG4-RD
  • 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

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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