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  • Neuropathic Pain in NMOSD Reduces Physical and Mental Wellness
    IgG4-RD history
  • Romiplostim Is a Possible Alternative to Multiple Platelet Transfusions in NICU
    Platelets in blood
  • 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
  • As Awareness of Huntington Grows Through Help 4 HD and Others, Hope Is Building

Latest News

More News
  • Asthma May Be a Key Risk Factor for ACS in Patients With Sickle Cell Disease
  • Avacopan Effective in Patients With GPA and MPA With Kidney Involvement
  • Withholding Cyclophosphamide in Low-Risk EGPA Does Not Increase Relapse
  • Annual Rituximab Infusions May Provide Sustained Disease Control in AAV
  • Recommendations Updated for Managing AAV
  • Elastase Inhibitory Activity Linked to COPD Severity in Patients With AATD
  • Fetal Treatments for Severe HDFN Show Success in Slovenia
  • Phototherapy Modifies Hematologic Markers Without Inducing Inflammation
  • Physical Activity in Pregnancy Not Associated With Negative Outcomes
  • Long-Term Avapritinib Extends Survival and Relieves Symptoms in SM
  • Anemia, Interosseous-Intramuscular Bleeding Cause Delayed SM Diagnosis
  • Premature Menopause, Hormone Therapy Linked to Worse Hemodynamics in PAH

Latest Features

More Features
  • 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.
  • Muldoon family
    Pioneering Case Illustrates the Promise of Personalized Gene Editing for Rare Diseases
    By Manuela Callari, PhD
    KJ Muldoon has been achieving developmental milestones, but his long-term prognosis is unknown.

Conference Coverage

MORE MEETINGS
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WODC 2025

World Orphan Drug Congress USA

Latest Patient Perspectives

More Patient Perspectives
  • Learning How Medical Language Matters Through My CIDP Diagnosis
    Of course, getting sick with chronic inflammatory demyelinating polyneuropathy (CIDP) forced me to address why I felt this way.
    By Jaimie Sheil, CIDP Contributor
    June 16, 2025
  • MG Month Reminder: Advocacy Takes a Front Seat
    I want every patient who presents with myasthenia gravis (MG) symptoms to be heard.
    By Stefanie Shea-Akers, MG Contributor
    June 16, 2025
  • Friedreich Ataxia’s Impact on Driving
    Back then, I was more ambulatory, but I still had to focus on the impact Friedreich ataxia (FA) had on driving.
    By Lauren Gaczhias, FA Contributor
    June 16, 2025
  • The Hidden Costs of Living With Pompe Disease
    As someone living with Pompe disease, I'm all too familiar with the complexities of managing a rare genetic disorder.
    By Bruce Campbell, Pompe Disease Contributor
    June 16, 2025

Latest HCP Insights

More Insights
  • 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.
  • Asian patient with doctor
    Exploring the Use of Spesolimab in Managing Chronic GPP
    By Ryner Lai, MBBS
    The recent availability of spesolimab has transformed the treatment landscape for patients with chronic generalized pustular psoriasis (GPP).
  • prognosis
    Stratifying Risk and Predicting Prognosis in CLL
    By Ryner Lai, MBBS
    The ability of clinicians to stratify risk and predict prognosis in chronic lymphocytic leukemia (CLL) has improved by leaps and bounds.

Latest Rare Care Podcast

More Episodes
  • 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’
  • An Interview With Dr. Antonella Favit-Van Pelt on Neuromodulation to Treat MS
  • An Interview Lauren Holder, an Advocate and Patient With Huntington Disease

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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