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Alagille Syndrome
Understanding Alagille Syndrome with Noelle Ebel, MD
Prognostic markers in maralixibat treated ALGS may guide clinical decision-making
ALGS patients have nearly 1 medical visit per week
Autoimmune
ALP, bilirubin important biomarkers in managing primary biliary cholangitis risk
Primary biliary cholangitis disease course, prognosis vary greatly
Rapid prednisone tapering safe in patients with myasthenia gravis
Cardiovascular
Distinct macrophage subsets identified in pediatric cholestatic liver disease
Hypertension significantly more common in patients with multiple sclerosis
Study: More than 250K people expected to die from infective endocarditis by 2030
Gastroenterology and Hepatology
Hospitalization data provides new insight on healthcare utilization in Wilson’s Disease
Milk elimination diet improves symptoms, remission rates in children with Eosinophilic Esophagitis
$9.1M Awarded for Liver Cancer Research
Hematology
FDA approves first treatment for children and adolescents with PNH
FDA Grants DSG3-CAART Orphan Drug Designation for the Treatment of Pemphigus Vulgaris
FDA approves Empaveli for paroxysmal nocturnal hemoglobinuria
Interstitial Lung Disease
Nailfold videocapillaroscopy can increase diagnostic accuracy of ILD
FDA awards 11 grants for rare disease treatment development
ILD decreasing as cause of hospitalization, mortality in SSc
Lupus Nephritis
Nearly one-third of men with SLE develop serositis
Economic burden of lupus nephritis is substantial
‘Profound’ disparities in lupus nephritis mortality by race, urban residence
Metabolic
Recent-onset diabetes, weight loss increases risk for cancer
Blood glucose reduced with cinnamon supplement in patients with prediabetes
New evaluation measure announced for kidney disease testing in people with diabetes
Musculoskeletal/Rheumatology
Cognitive behavioral therapy not as good as NSAIDs for arthritis pain
IV immunoglobulins plus methylprednisolone improves fever course in MIS-C
Ehlers-Danlos syndrome associated with GI, cardiovascular symptoms
Nephrology
Optimizing Electronic Health Record Data for Diagnosing Rare Diseases
Combination Therapy Demonstrates Clinically Meaningful Efficacy in RCC
Terlipressin improved renal function in patients with hepatorenal syndrome
Neurology
AMX0035 lower long-term risk of death in ALS
First symptoms predict disease progression in Metachromatic Leukodystrophy
Investigational treatment prolongs survival in ALS
Oncology
Metabolic syndrome may be an important risk factor for HCC
Durvalumab, Tremelimumab Granted Orphan Drug Status for Liver Cancer
Vemurafenib plus rituximab associated with durable complete response in HCL
Ophthalmology
Usher Syndrome: A Patient Perspective with Molly Watt
Clinical Trials for Retinal Disease Gene Therapies is Driving the Need for Genetic Testing
Statins improves Graves’ orbitopathy outcomes in patients with active eye disease
Pulmonary and Lung
Have you switched PAH patients in the inpatient setting from inhaled tresprotinil to alternatives due to COVID-19 risks?
Improvement in non-invasive measurements seen after tadalafil in children with PAH
Tadalafil may fulfill unmet need in pediatric patients with PAH
Sarcoma
Pazopanib, trabectedin, or eribulin? Can immune-related markers dictate which to use in STS?
Wound complication rates lower in sarcoma patients at academic centers
Pandemic greatly reduced access to musculoskeletal cancer care
Thyroid Eye Disease-ASOPRS
Recalcitrant TED responsive to teprotumumab
Case Series: TED reactivation after COVID-19 vaccination
How did pandemic-related teprotumumab treatment delays impact outcomes?
Infectious Diseases
Study examines risk estimation of sexual transmission of Zika virus
Calcium channel blockers decrease the risk of active tuberculosis
Combination treatment reduces mortality in hospitalized patients with MERS
Genetic and congenital
FDA Approves First Therapy for Children with Neurofibromatosis Type 1
Pediatric bone marrow failure may be linked to underlying primary immunodeficiency
Niemann pick disease treatment granted fast track designation
Growth Hormone Disorders
Alert system helps clinicians monitor adherence to rhGH treatment
Heights similar between patients with isolated GH deficiency, idiopathic short stature
Early diagnosis essential to improving outcomes in rare diseases affecting growth
Thyroid Eye Disease-AAO
QUIZ: Understanding the Natural History of Thyroid Eye Disease
Teprotumumab adherence rates are high in patients with TED
Teprotumumab-related adverse events common but mild
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Case Study Quiz: Lupus Nephritis
Quiz
Case Study Quiz: Lupus Nephritis
Posted on June 16, 2021
<section><h2>[COPY] [COPY] Case Study Quiz: Lupus Nephritis </h2><p></p></section><section><h2><p>A 42-year-old male patient with lupus nephritis class IV diagnosed 4 years ago, progressed to ESRD after failing multiple immunosuppression therapy. He had an AVF placed in his left forearm that is maturing well with a plan to start hemodialysis in the next few weeks.Patient is very disappointed with the outcome. </p><p> </p><p><strong>Which of the following factors has contributed to the poor progression?</strong></p></h2><p></p></section><section><h3><p><strong></strong><strong>What should the patient expect after starting hemodialysis?</strong></p></h3></section><section><h3><p>The patient is interested in transplant evaluation. </p><p> </p><p><strong>Which is the correct statement regarding kidney transplant in lupus patients?</strong></p></h3></section><section><h2></h2></section><section><h3></h3></section>
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