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  • Long-Term Avapritinib Extends Survival and Relieves Symptoms in SM
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  • Rilzabrutinib Reduces Flares in IgG4-RD, Phase 2 Study Shows
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  • Emerging Solutions Could Make TK2d Easier to Recognize and Treat
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  • 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
  • FDA Grants Orphan Drug Designation to VAS-101 for Sickle Cell Disease
  • Patients With PsA and GPP May Be at Risk of Severe Disease and a Poor Prognosis
  • A Case of GPP After Steroid Withdrawal in a Patient With Breast Cancer
  • Modern Long-Term Prophylaxis Is Reshaping HAE Management
  • Lanadelumab Dosing Interval Extension Retains Effectiveness to Reduce HAE Attack Rates
  • Novel Agents Outperform Traditional Therapies in Older Adults With CLL
  • Cold-Induced Bradykinin Measurement a Useful Biomarker in HAE
  • Spesolimab Shows Promise for Treating GPP During Dialysis
  • Methylphenidate Reduces Fatigue in Hematologic Malignancies, MDS
  • New Study Identifies Elevated mCRP as a Marker of AAV Activity
  • Muscle Biopsy Can Distinguish AAV Muscle Vasculitis From Localized Muscle Disease
  • Abatacept Achieves Disease Control in Refractory Tracheobronchial GPA

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
  • 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
  • A Pompe Turning Point: Needing a Wheelchair
    I went years without being on Pompe treatment, and walking became my most noticeable struggle.
    By Keyana Sullivan, Pompe Disease Contributor
    June 24, 2025
  • pompe disease complications
    Ways to Make Life With Pompe Easier 
    The most common challenge that comes with living with Pompe disease is getting tired more easily.
    By Keyana Sullivan, Pompe Disease Contributor
    June 24, 2025

Latest HCP Insights

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  • 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.
  • Medical questionnaire
    Exploring the Factors That Impact Prognosis in NMOSD
    By Ryner Lai, MBBS
    An early age of disease onset and the early initiation of treatment are both associated with an improved prognosis in NMOSD.
  • Diagnosis
    Diagnosis of CIDP Revised to Leprosy Neuropathy
    By Ryner Lai, MBBS
    A patient initially received a diagnosis of CIDP, but it was later reviewed to leprosy neuropathy.

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 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
  • Test Your Knowledge About the Etiology of Achondroplasia

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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