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Rare Diseases

ANA Pollution (2.06.2026)

Dr. Jack Cush reviews the news, journal articles and regulatory news from this past week on RheumNow.com

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FDA vs. Amgen: At Odds Over Avacopan

MedPage Today

The FDA has requested that Amgen voluntarily withdraw avacopan (Tavneos), a treatment for anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis, but the company said it has no plans to pull the drug from the market.

Originally developed by ChemoCentryx, the oral

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Only a subset of incomplete lupus (ILE) will progress to SLE - betw 1 in 10 and 1 in 5. Can you Prevent SLE? Dr. David Karp lecturing at UTSW @drdavidkarp @utswrheum https://t.co/oo40Dumk4I
Dr. John Cush @RheumNow( View Tweet )

Giant Cell Arteritis Outcomes in Canada

A retrospective cohort study of patients with giant cell arteritis (GCA) demonstrates relapses are common and seen in nearly half of patients, were common after treatment is stopped and is not effectively averted by methotrexate (MTX).

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Aortitis in Giant Cell Arteritis Treated with Tocilizumab

The The Tocilizumab in Giant Cell Arteritis Spanish Collaborative Group studied giant cell arteritis with aortitis, comparing the efficacy of intravenous vs. subcutaneously (SC) tocilizumab (TCZ) - demonstrating the superiority of SC TCZ.

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B Cells at the Brink in Sjögren Disease The current issue of Arthritis & Rheumatology reviews the current understanding of the immunopathogenesis of Sjögren disease (SjD) and how this understanding has informed drug development for this systemic autoimmune disorder. https://t.co/zI7lQ5N54o
Dr. John Cush @RheumNow( View Tweet )

DMARD Responses in Localized Scleroderma

JAMA Dermatology has published the results of a juvenile localized scleroderma (JLS) trial showing that mycophenolate mofetil (MMF) and methotrexate (MTX) are equally effective in treating JLS, noting low flare rates and possibly better tolerability with MMF.

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🧬 PAPA Syndrome: When Sterile Inflammation Mimics Infection (Pyogenic Arthritis, Pyoderma gangrenosum, Acne) •A rare monogenic autoinflammatory disease •Caused by gain-of-function mutations in PSTPIP1 🧠 Pathophysiology •Mutant PSTPIP1 → increased interaction with pyrin https://t.co/tJ4INdgfky
Ravi Kumar @RheumattDoc( View Tweet )
What Is Restless Legs Syndrome? JAMA Patient Education handout. https://t.co/fCqArVxrSu https://t.co/WjFNWyr9Q2
Dr. John Cush @RheumNow( View Tweet )

Emergencies, Independence & Hemorrhage (1.23.2026)

Dr. Jack Cush reviews the news and reports from the past two weeks on RheumNow.Com

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Congratulations to Lupus Research Alliance 2025 Global Science Award Recipients 1) Drs. Deepak Rao & Alexandra-Chloe Villani at Harvard affiliates; & 2) Dr. Caroline Jefferies at Cedars-Sinai. Grants will support interdisciplinary, collaborative, & synergistic projects in SLE https://t.co/GcfuEty75b
Dr. John Cush @RheumNow( View Tweet )
Spread of Psoriatic Disease from Skin to Joints Approximately 20-30 % of all people who suffer from psoriasis also develop painful inflammation in their joints over time. If left untreated, PsA can lead to permanent damage to bones and joints. Why the disease progresses was https://t.co/UbGHFASKvc
Dr. John Cush @RheumNow( View Tweet )
Anti-mitochondrial M2 Ab (AMA-M2) in 987 IIM pts-- 55 (6%) were AMA-M2 positive. AMA-M2+ had signif. higher rates of polymyositis (56% vs 23.5%), GGT, alk phos levels, & 3 fold more cardiac involvement (60 vs 13%) (arrhythmias, CHF, pulm HTN (31-56%), but no effect on survival https://t.co/8fQzgRfHr4
Dr. John Cush @RheumNow( View Tweet )
CASTLE Phase 1/2a CAR-T cell trial of 24 Tx refractory SLE, SSC, IIM pts w/ MB-CART19.1 (autologous CD19 CAR-T). Good safety (no CRS Gr 2 or higher or ICANS) @wk 24 & 22/24 achieved efficacy (9/10 SLE remission; 9/9 SSc no progression, 4/5 IIM major/mod response). All GC free on https://t.co/1BJidCuCeb
Dr. John Cush @RheumNow( View Tweet )
Anti-mitochondrial M2 Ab (AMA-M2) in 987 IIM pts-- 55 (6%) were AMA-M2 positive. AMA-M2+ had signif. higher rates of polymyositis (56% vs 23.5%), GGT, alk phos levels, & 3 fold more cardiac involvement (60 vs 13%) (arrhythmias, CHF, pulm HTN (31-56%), but no effect on survival https://t.co/WYWeDpiYOh
Dr. John Cush @RheumNow( View Tweet )
Best of 2025: ERS/EULAR guidelines for CTD-related interstitial lung disease The ERS and EULAR have published clinical practice guidelines for the evaluation and management of CTD associated ILD in two simultaneous publications in the European Respiratory Journal and Annals of https://t.co/ceGCQjkmJV
Dr. John Cush @RheumNow( View Tweet )
Sjogrens is a hot area in drug development: “Sjogren’s Syndrome Pipeline Insight 2025” report provides comprehensive insights about 20+ companies and 22+ pipeline drugs, including: nipocalimab VAY736 (ianalumab) HZN-1116 AMG 329 R-2487 https://t.co/sVbyPLi2Um https://t.co/2URpGUsMk9
Dr. John Cush @RheumNow( View Tweet )
Best of 2025: Glossary for Giant Cell Arteritis Dejaco and colleagues have published a glossary of terms often used in conjunction with giant cell arteritis. The glossary of definitions for 23 signs and symptoms of GCA was developed through a consensus process involving https://t.co/yfJXZx5r6G
Dr. John Cush @RheumNow( View Tweet )

Best of 2025: ERS/EULAR guidelines for CTD-related interstitial lung disease

The European Respiratory Society (ERS) and European Alliance of Associations for Rheumatology (EULAR) have published clinical practice guidelines for the evaluation and management of connective tissue diseases (CTD) associated interstitial lung disease (ILD) in two simultaneous publications in

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Rheumatologists can’t ignore hypereosinopilic syndrome anymore, not when it borders on our diseases and therapies. Great takeaways on subtypes and therapies from Giacomo Emmi in an elegant talk #EULAR2025 https://t.co/yzQVt1fLKW https://t.co/hrbEeppZhW
Dr. John Cush @RheumNow( View Tweet )

Best of 2025: Dermatomyositis Reviewed

Researchers from the University of Pennsylvania have published an open-access review of dermatomyositis (DM) - its manifestations, hallmark cutaneous features, etiopathogenesis and treatment options.

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Disease Activity Criteria for Adult-Onset Still's Disease: EULAR Points to Consider EULAR has published a "points to consider" (PtCs) guidance on the development of criteria for the assessment of the disease activity in adult-onset Still’s disease (AOSD). https://t.co/3FikFiGgxM
Dr. John Cush @RheumNow( View Tweet )

Nerandomilast FDA Approved for Progressive Pulmonary Fibrosis

The FDA has approved nerandomilast (Jascayd), a PDE4b inhibitor, to treat adults with progressive pulmonary fibrosis (PPF). Nerandomilast was approved to treat idiopathic pulmonary fibrosis (IFP) in October 2025.  

PPF is a a life-threatening, progressive lung condition, leading to

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Best of 2025: Overview of the VEXAS Syndrome

A current systematic review in Rheumatology addresses the clinical features seen in the VEXAS syndrome.

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Telling You Where to Go (12.19.2025)

Dr. Jack Cush reviews the news and journal articles from the past week on RheumNow.com. Who would've thunk - a PsA FMF connection? Labs to diagnose SSc-ILD? IgA-RF may be important or RZV may not work in JAK inhibitor treated patients?

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