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Article

New, Improved Therapy for Pompe Disease

Jack Cush, MD

Researchers from the University of Cincinnati Gardner Neuroscience Institute will present data on a new treatment for Pompe disease at the American Academy of Neurology on April 20th; the results show that treatment with avalglucosidase alfa yielded substantial clinical improvements and a more favorable safety profile compared to the current standard of care (alglucosidase alfa).

Article

Autoimmune Risk of Death Up during COVID

Jack Cush, MD

A population-based study in the UK shows that patients with rare autoimmune rheumatic diseases (RAIRD) were 44% more likely to die during the 2020 COVID-19 pandemic (March - April) compared to either the general population or their pre-COVID risk.

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KDAO2011
Dr. R Fleischmann "Don't be afraid to use TOFA!" FDA warnings on⬆️ MACE/malign based on 1 study (Pfizer 1133) in high risk patients. Older age, tobacco use are risks for MACE/CV events and cancer. Meta-analyses, registries, clinical trials program do NOT show⬆️ risk #RNL2021 https://t.co/4p9YqyDrGz
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Randomized trial of 206 adults with knee OA found that a web-based exercise intervention supported by text messaging improved knee pain and function at 24 weeks vs web-based information alone. (MCID pain 72.1% vs 42%) https://t.co/h5UaedS4Zm
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RheumNow
Not in the US, but biosimilar infliximab (CT-P13) is being used subcutaneously in the EU and Canada. Analysis of 2 RCTs shows (compared to IV inflectra or remicade) SC CT-P13 greater DAS28-CRP, CDAI and SDAI response rates. https://t.co/prSVJ0BIdd
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RheumNow
Scoot over small molecule inhibitors, the future might be with nanoparticle targeting for RA! Dr. Fleischmann reviews study on nanoparticle targeting of CCP Ab that showed noninferiority to Tocilizumab #RNL2021 coverage from @KDAO2011
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#RNL2021 Coverage from Dr. Rachel Tate @uptoTate The good and the bad of HLA B27 iritis from Dr. Wang #RNL2021 -- 90% of patient maintain 20/40 vision or better with treatment -- BUT disease can relapse and can alternate between eyes
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Johns Hopkins myositis clinic: anti-mitochondrial Abs (AMA) found in 5% adult myositis, 1% of juv. myositis pts. AMA assoc. w/ severe muscle weakness, Raynauds, dysphagia, cardiomyopathy & treatment refractory Dz - Screening for cardiac & swallowing https://t.co/oj9kwHgTHf
Dr. John Cush Tweet
RheumNow
ASSAIL-MI trial studied 200 pt with new (<6hr) STEMI-randomized to either PBO or IV 280 mg tocilizumab. While no signif difference in final infarct size (7.2 vs 9.1%; P=.08), TCZ treated pts had better myocardial salvage & less Microvascular obstruction https://t.co/WRRGXCbP5O
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RheumNow
Non-Hallmark manifestations of dermatomyositis include: panniculitis, diffuse subcutaneous edema, erythroderma, calcinosis, ulceration, flagellate erythema, Wong-type DM, gingival telangiectasias, and the ovoid palatal patch (full read) https://t.co/x6cdP3WDcW
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