Case Report: Bilateral Total Hip Arthroplasty in a Patient With Achondroplasia
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National Amyloidosis Awareness Month: Expert Says Future of Treatment Is BrightAmyloidosis, which has several types, is diagnosed in at least 4000 Americans per year. -
More PAH Therapies Than Ever Before, Yet Time to Diagnosis Hasn’t ImprovedThe number of treatments for PAH continues to rise, but it still takes an an average of 3 years from symptom onset to diagnosis. -
Colorado-Based ‘Can Do MS’ Helps People With Multiple Sclerosis Live Healthier LivesCan Do MS provides free programs on topics such as nutrition, exercise, MS symptoms, relationships, and employment. -
Smoking and Air Pollution May Exacerbate MDS and Shorten LifespanBlack carbon and PM2.5, toxins with known links to cancers, correlated highly with MDS disease severity scores in a New York study. -
Rare Disease Doctors Tout Value of Social Media While Warning of Online MisinformationThe average American has 7.1 social media accounts, according to industry statistics.
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A Different Disability Accommodation That Resonates With Me as a Patient With FAFriedreich ataxia (FA) is a constant challenge.March 13, 2025 -
A Beacon of Hope Found for the Future of Pompe Disease TreatmentAfter a decade of searching for an answer to what was causing progressive pain and weakness in my body, I was finally diagnosed with Pompe disease.March 13, 2025
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How Cold Agglutinin Disease and UTIs OverlapWhen you have a rare disease such as cold agglutinin disease (CAD), you need to be diligent to avoid infections.March 12, 2025 -
How My Liver Biopsy Turned Into a NightmareI do not want to discourage other primary biliary cholangitis (PBC) patients from using this procedure.March 11, 2025
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Managing Concurrent HDFN and FNAIT: A Rare CaseA neonate with known HDFN was discovered to also have FNAIT after birth, necessitating immediate, intensive intervention. -
Momelotinib May Be a Promising Treatment Option for Patients With Myelofibrosis-Associated AnemiaMyelofibrosis-associated anemia can often result in significant levels of fatigue, impairing health-related quality of life. -
Patients With ATTR-CM May Benefit From Early Screening and DiagnosisOne of the most effective ways of improving outcomes is the implementation of early screening and diagnosis of ATTR-CM. -
What Is the Relationship Between IgG4-RD and Autoimmune Disease?Research suggests there is some relationship between IgG4-RD and autoimmune rheumatic diseases that deserves further exploration. -
Current and Emerging Strategies for Managing AchondroplasiaThe management of achondroplasia involves several strategies, including the first approved precision medicine, vosoritide.
- 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
- Fetal and Neonatal Alloimmune Thrombocytopenia
- Friedreich Ataxia
- Gastrointestinal Stromal Tumor
- Generalized Pustular Psoriasis
- Hemolytic Disease of the Fetus and Newborn
- Hemophilia
- 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
- Transthyretin Amyloid Polyneuropathy
- Transthyretin-Mediated Amyloid Cardiomyopathy
- Wilson Disease
