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  • Romiplostim Is a Possible Alternative to Multiple Platelet Transfusions in NICU
    Platelets in blood
  • New Study Suggests Serum IgG4 Levels Not Perfect Tool to Diagnose IgG4-RD
    Blood serum testing
  • 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
  • Pioneering Case Illustrates the Promise of Personalized Gene Editing for Rare Diseases
    Muldoon family

Latest News

More News
  • Focal Radiotherapy May Extend Survival in Primary CNS Lymphoma
  • Transaminases Offer Limited Prognostic Value in Hepatic WD
  • Column Agglutination Technique Better Predicts Neonatal HDFN Outcomes
  • Bilirubin Concentrations Can Vary Between Different Ethnicities
  • Patients With SCD Exhibit an Elevated Cardiovascular Disease Burden
  • Evaluating HDFN Risk With “Confusing” Blood Group Antibodies, Such As Anti-G, Anti-M
  • AATD Remains Underdiagnosed in Pre-Liver Transplant Candidate Evaluations
  • Study Reveals Distinct Outcomes Across Systemic Mastocytosis Subtypes
  • Detecting C-KIT Mutations Helps Diagnose Concurrent SM and AML
  • New LC-MS/MS Assay Enhances Detection of Systemic Mastocytosis and HαT
  • Patients With CIDP May Benefit From Fall Risk and Walking Aid Assessment
  • Some Plasma Biomarkers Have Ability to Predict Outcomes in PAH

Latest Features

More Features
  • 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.
  • 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.
  • Victory Over Vasculitis group members
    Vasculitis Awareness Month Highlights Advances in Treatment and Ongoing Obstacles
    By Larry Luxner
    The nonprofit Vasculitis Foundation advocates for patients with 1 or more of the 20-plus subtypes of the disease.
  • 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.

Conference Coverage

MORE MEETINGS
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WODC 2025

World Orphan Drug Congress USA

Latest Patient Perspectives

More Patient Perspectives
  • Maintaining Strength With AATD
    Though muscle weakness may not be caused by alpha-1 antitrypsin deficiency (AATD), maintaining strength is important for quality of life.
    By Alyssa Gloor, AATD Contributor
    June 13, 2025
  • Balancing AATD and Housecleaning Chores
    Individuals with alpha-1 antitrypsin deficiency (AATD) may need to clean their environment more thoroughly for safer breathing.
    By Alyssa Gloor, AATD Contributor
    June 13, 2025
  • Woman hiking
    Reflecting on My Life Before Indolent Systemic Mastocytosis
    Even though I was battling indolent SM, I was constantly on the go and finding new things to do. I was mentally prepared for flare-ups.
    By Tara Keith, SM Contributor
    June 13, 2025
  • DMD history
    How I Manage Patient Burnout
    Living with LGMD, there have been plenty of times when I’ve had to deal with healthcare workers not taking my health seriously.
    By Jakira Avery, MD Contributor
    June 12, 2025

Latest HCP Insights

More Insights
  • 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.
  • Doctor holding clipboard
    Ensuring the Optimal Management of Myelofibrosis-Associated Anemia
    By Ryner Lai, MBBS
    The burden of care for myelofibrosis-associated anemia is high, especially among patients who are transfusion-dependent.
  • fetus and placenta
    The Role of Inflammation as a Key Driver in FNAIT
    By Ryner Lai, MBBS
    A recent study indicated that placental inflammation may be a key disease modulator in fetal and neonatal alloimmune thrombocytopenia.
  • Newborn screening
    When Is Genetic Testing Appropriate to Investigate a Diagnosis of HAE in Children?
    By Ryner Lai, MBBS
    A case study demonstrates how genetic testing can be useful in children with a positive family history of hereditary angioedema.
  • palliative care
    Enhancing Palliative Care in HCC
    By Ryner Lai, MBBS
    Palliative care should be integrated into the early treatment and management of patients with hepatocellular carcinoma (HCC).

Latest Rare Care Podcast

More Episodes
  • 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
  • An Interview With Joyce Kullman, Executive Director of the Vasculitis Foundation

Quizzes

More Quizzes
  • 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
  • Test Your Knowledge About the Etiology of PAH

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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