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Alagille Syndrome
Allucent launches decentralized clinical trial platform at 2023 World Orphan Drug Congress
Case study explores off-label use of odevixibat in ALGS
Regulatory Approval and Commercialization: Developing Treatments for Rare Diseases
Autoimmune
Methotrexate/prednisone combo may be effective in alopecia areata totalis, universalis
FDA approves sutimlimab for cold agglutinin disease
ALP, bilirubin important biomarkers in managing primary biliary cholangitis risk
Cardiology
Nearly half with high BP maintain long-term absence of coronary artery calcium, significantly lowering cardiovascular risk
Adolescent hypertension linked with increased risk of stroke in young adulthood
Clopidogrel is better than aspirin after coronary stenting in patients with diabetes
Endocrinology
Pulmonary function in patients with type 2 diabetes worse than health population
How does thyroid eye disease impact buccal fat pad volume?
Higher dose, earlier start to GH therapy can lead to greater growth response
Gastroenterology
Administering simethicone on same day as colonoscopy maximizes benefits
Reengagement of HCV patients lost to follow-up needed to achieve WHO goals
Vedolizumab found safe, effective in post-marketing surveillance
Genetic and Congenital
Continued use of burosumab in X-linked hypophosphatemia needed to sustain clinical benefit
First patient enrolled in pediatric clinical trial of leniolisib for APDS
FDA accepts priority review for leniolisib for treatment of APDS
Growth Hormone Disorders
Outcomes similar between middle-aged, older patients with growth hormone deficiency after GHRT
Growth hormone replacement therapy safe in cancer survivors with growth hormone deficiency
Long-term use of rhGH replacement therapy improves bone mineral density, body composition
Hematology
FDA approves avapritinib for indolent systemic mastocytosis
Comorbid Alzheimer’s disease is a risk factor of acquired hemophilia A incidence
Rare disease patients should be considered ‘high-value managers’ of their condition
Industry News
Ipsen to acquire Albireo accelerating growth in rare disease with treatments for several pediatric liver diseases
Amgen to buy Horizon Therapeutics
Alexion, AstraZeneca Rare Disease to Acquire LogicBio Therapeutics to Accelerate Growth in Genomic Medicine
Infectious Diseases
FDA Approves First Respiratory Syncytial Virus (RSV) Vaccine
New study suggests link between chronic kidney disease, in-hospital mortality in COVID-19 patients
RSV vs Influenza: Which causes more severe respiratory illness in children under 2-years-old?
Inherited Retinal Diseases
Study: Average delay to reach a correct inherited retinal disease diagnosis is 15 years
Study explores motivations for pursuing ocular genetic testing
Study reveals the most common inherited retinal diseases globally and regionally
Internal Medicine
Administering simethicone on same day as colonoscopy maximizes benefits
Efficacy, adverse effects of gefapixant are dose-dependent in chronic cough treatment
Non-invasive electromagnetic therapy shows promise in rapid unspecific back pain relief
Interstitial Lung Disease
Pulmonary arterial hypertension in combination with ILD associated with worse overall prognosis
Lung ultrasound has potential to detect interstitial lung disease
Single molecule Molecular Inversion Probes may be more cost-effective in some IRDs
Lupus Nephritis
Lupus nephritis linked with higher infection-related hospitalization in young adults with SLE
Study describes PFT abnormalities in childhood-onset systemic lupus erythematosus
Treatment response at 6 months may predict poor long-term renal outcomes in lupus nephritis
Managed Markets
Real-time benefit tools for medication costs wanted by older adults
Most patients want to see test results in patient portal as soon as possible
Intensive blood pressure management cost-effective in older adults
Metabolic
What All Endocrinologists Should Know about Cushing disease
Recent-onset diabetes, weight loss increases risk for cancer
Blood glucose reduced with cinnamon supplement in patients with prediabetes
Nephrology
New study suggests link between chronic kidney disease, in-hospital mortality in COVID-19 patients
Children with low birth weight have a higher rate of reduced kidney function than previously reported
Continued use of burosumab in X-linked hypophosphatemia needed to sustain clinical benefit
Neurology
Longer training at vigorous intensity may be best for patients with chronic stroke
Women with Parkinson’s disease may have worse gastrointestinal symptoms than men
COVID-19 vaccination doesn’t seem to increase risk of relapse in multiple sclerosis
Oncology
Significant discrepancies found in reporting of protocol eligibility criteria changes in oncology clinical trials
Bacterial decolonization reduces acute radiation dermatitis prophylaxis
Positive outcomes possible for patients who stop immune checkpoint inhibitor treatment
Ophthalmology
Study: Average delay to reach a correct inherited retinal disease diagnosis is 15 years
Thyroid eye disease shows unimodal age distribution, peaking at younger ages in certain races
Study explores motivations for pursuing ocular genetic testing
Progressive Familial Intrahepatic Cholestasis
Case study explores off-label use of odevixibat in ALGS
Regulatory Approval and Commercialization: Developing Treatments for Rare Diseases
Ipsen to acquire Albireo accelerating growth in rare disease with treatments for several pediatric liver diseases
Pulmonology
Efficacy, adverse effects of gefapixant are dose-dependent in chronic cough treatment
Pulmonary function in patients with type 2 diabetes worse than health population
Pulmonary vein stent implantation effective as first-line therapy for pulmonary vein stenosis
Rheumatology
Rheumatoid arthritis increases risk of DED, corneal surface damage
FDA approves abaloparatide for osteoporosis in men
Rheumatoid arthritis incidence rate increasing worldwide
Sarcoma
Does treatment at sarcoma centers improve prognosis in STS?
Standardized surgical plan for fibular proximal malignant tumors needed to improve outcomes
Implantable venous access devices outperform PICCs in cancer treatment
Thyroid Eye Disease
Thyroid eye disease shows unimodal age distribution, peaking at younger ages in certain races
Severe meibomian gland dysfunction linked to tear film instability in thyroid eye disease
IRMPT for strabismus repair in TED results in durable relief of diplopia
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Ronald M. Laxer, MDCM, FRCPC, addresses rheumatologic disease ‘masquerading’ as infection
Infectious Diseases
Musculoskeletal/Rheumatology
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Ronald M. Laxer, MDCM, FRCPC, addresses rheumatologic disease ‘masquerading’ as infection
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