Participate in Research

3:00The 2024 Chapel Hill VF Conference
Vasculitis Research: What’s New, What’s on the Horizon
Presenter: Vimal Derebail, MD

0:00
3:00 What’s Old? Former standard treatment approach with corticosteroids.
5:18 Approaches to disease management for ANCA vasculitis.
5:27 Steroids in ANCA vasculitis.
7:37 Reduced steroid doses in ANCA vasculitis.
10:10 Complement in ANCA vasculitis.
11:45 Avocopan update
12:47 Duration of maintenance therapy.
14:50 Long-term remission off therapy.
17:05 Here's what is on the horizon.
20:19 The PEXIVAS Study.
21:01 How do we study rare diseases?
22:31 Collaberative care in vasculitis.
23:49 Looking at delays in diagnosis.
26:31 Summary.

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Emerging Cellular Therapies
Presenter: Dr. Meghan Free
Chapel Hill 2024 Regional Conference

0:00
3:44 Types of blood cells.
4:34 A lineup of lymphocytes.
6:23 Important immune cell types in ANCA vasculitis.
7:53 Long-term Remission Off Therapy (LTROT)
9:11 What is “immunological remission”?
11:04 What do we know so far about LRTOT?
13:00 Specialized, antigen, or cell-specific therapies.
14:52 Targeted therapy over broad immunosuppression.
15:39 Chimeric Antigen Receptor (CAR-T Cells).
18:43 Pros and cons with CAR-T cell therapy.
21:53 Regulatory T-Cells. (Tregs)
22:56 What do we know about Tregs in ANCA vasculitis.
27:24 Can we try to restore Treg function in patients?
30:38 Summary of presentation.

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0:00
0:42 Introducing Dr. Sebastian Sattui.
1:32 Why this meeting is so important.
3:06 The MANDARA Clinical Trial
3:46 The TAPIR Study
5:07 Honoring Dr. Ronald Falk
6:29 Using renal biopsy info or urinary markers to predict how treatments will go for
renal function.
6:59 Results from the VascStrong Study.
9:21 Presentation on AVOCOPAN.
11:25 Excitement around the new response criteria being developed around ANCA vasculitis.
12:23 Focusing on multi-disciplinary care.
14:29 Joyce Kullman: The International Patient Meeting.
16:47 Translating educational materials into other languages.
17:36 Q: Were there any sessions dealing with treatment for PAN?
20:07 Q: Was there a discussion about revisions to the use and timetable with steroids?
22:06 Q: Could you give some more detail about the MANDARA study?
24:48 Q: What would you suggest so that our doctors and medical providers get the information
that you are sharing today.
27:32 Q: Was there any discussion about the use of artificial intelligence technology in vasculitis
research?

2024 Barcelona Vasculitis Workshop: Research Highlights
Recorded May 2024

More than 900 medical professionals from 50 countries came together in Barcelona last month for the 21st International Vasculitis Workshop. Their purpose was to focus on vasculitis and related disorders.
In this webinar, Sebastian Sattui, MD, MS, Director of the UPMC Vasculitis Center in Pittsburgh, shares some of the notable research news, developments, and promising developments presented at the meeting.

Among the studies he discusses:
· MANDARA Study (benralizumab vs mepolizumab) for EGPA
· New data (real life and post hoc analyses) about the efficacy and tolerance of avacopan in AAV

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0:00
0:28 Introducing Dr. Mike Putman
2:07 Explaining the PEXIVAS Trial
3:12 What is a Randomized Controlled Trial (RCT)?
6:16 What is plasma exchange?
9:14 A closer look at the demographics of participants in the PEXIVAS trial.
10:25 Primary outcomes from this study.
13:33 What did the primary outcomes show about infections?
14:24 Limitations of the PEXIVAS study.
17:39 Plasma exchange and insurance coverage.
19:04 Explaining this sentence: “The primary outcome is the time to the composite of all-cause
mortality and end-stage renal disease.”
21:15 Explaining this sentence: “Additional potential beneficial effects of PLEX in AAV include
removal of other mediators of inflammation and coagulation and effects on
immunoregulation.
22:30 Is the PEXIVAS study still recruiting?
24:03 How the PEXIVAS study is a model for international research collaboration.

Overview of the PEXIVAS Study
Recorded May 2024

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Welcome and Introductions. 0:01
Changes and Challenges by Jason Wadler. 2:31
How did your vasculitis affect your children? 15:21
Did you regain more than what you lost from your vasculitis? 17:35
First poll: What is the number one factor that affects long-term survival of vasculitis in men? 20:04
Vasculitis Impact on Men’s Health by Dr. Peter Grayson 21:06
Forms of vasculitis 24:34
Do some forms of vasculitis affect men more than women? 25:48
VEXAS Syndrome: A new form of vasculitis that only affects men! 27:42
Vasculitis and Survival. 31:31
Survival in women has improved more than men with vasculitis. Why? 32:28
Fertility issues and vasculitis for men. 36:34
COVID-19 and vasculitis. 38:33
Q: What is risk for patients with vasculitis to have surgery when infection is a major factor? 41:18
Q: What about testicular pain in men who have vasculitis? 44:36
Q: How can patients get in touch with you to be screened for VEXAS Syndrome? 46:07
How to be a Weekend Warrior with Vasculitis. By Brandon Hudgins and Art Diaz. 47:05
Exercising with vasculitis. 50:13
Art Diaz’s Testimonial. 58:18
Q: Do you build your exercise around your medication schedule? 1:10:53
Poll: What is primary reason men on average don’t participate in research? 1:14:21
Researchers Need More Men! by Dr. Peter Merkel and Paul Brown. 1:15:21
What’s the problem with men and research? 1:20;12
Paul Brown: Why I participate in research for vasculitis. 1:23:25
Q: Does your disease have to be active or inactive to participate in research? 1:37:20
Dealing with the Emotional Impact of Vasculitis: by John Stadler and Dan Bleicher. 1:40:40
John and Dan talk impact of vasculitis on their careers. 1:50:12

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0:00 Welcome and introductions.
1:36 Overall impression of the ACR 2023 Convergence.
2:59 Study on pregnancy outcomes with patients who have vasculitis.
4:28 The Great Debate: Using advanced therapies for patients with GCA and PMR.
6:28 The use of TAVNEOS® (avacopan) has everyone’s attention.
8:07 Excellent sessions on IgA Vasculitis.
11:21 Presence of patients at the ACR.
12:45 ACR addressing recruitment challenges in the rheumatology medical field.
15:53 Why are doctors and researchers excited about CAR T Cell Therapy?
Recorded: December 2023
Presenter: Sebastian Sattui, MD

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0:00 Welcome and introductions.
1:30 Great interest in steroid-sparing therapies.
2:18 Research into drugs like Rituxin that target B Cells.
2:57 EGPA getting greater focus in research.
4:18 Research into VEXAS.
5:39 Outcome criteria explained.
6:39 Explaining JAK-inhibitors.
7:35 Explaining CAR T Cell Therapy.
8:34 COVID discussion at the ACR

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0:00
1:04 Advances in treatment research and treatment of GPA/MPA.
2:20 New 2022 EULAR/ACR classification criteria.
3:51 Induction treatment of ANCA-associated vasculitis.
5:48 Glucocorticoid-sparing regimens.
8:00 Avacopan
9:11 Rituximab vs. azathioprine for maintenace of remission.
9:50 Spotlighting B-cell targeted therapy.
12:14 Advances in treatment and research for EGPA.
12:36 Clinical presentation of EGPA.
14:59 Classificaton critera for EGPA.
16:52 Treatment of EGPA.
20:05 Advances in treatment and research of LLV, and Polymyalgia Rheumatica
21:12 EULAR recommendations for the use of imaging in large vessel vasculitis.
22:20 Latest research into Polymyalgia Rheumatica.
24:10 Effect of Tocilizumab on disease activity in patients with active PMR.
26:51 Have you found there are discrepancies between the EULAR and ACR findings and recommendations?

Report from EULAR Advances in Research and Treatments
Recorded July 2023

Drs. Cornec, Berti, and Moura look at EULAR/ACR classification criteria and the demographics for vasculitis, as well as advances in research and treatments of GPA, MPA, EPGA, LVV, and PMR.  Diagnostic techniques are presented along with an overview of therapies including induction treatment, glucocorticoid-sparing regimens, plasma exchange, avacopan, rituximab versus azathioprine, B cell targeted therapy, personalized medicine, and more.

Expand your knowledge of vasculitis. Visit the Vasculitis Foundation website at: https://vasculitisfoundation.org/

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0:00
3:03 Why did you get involved in research?
10:29 Tell us what kind of research you do.

Meet our VCRC Fellows
Recorded July 2023

Dr. Byram introduces a few of the Vasculitis Clinical Research Consortium fellows including pediatric rheumatologist Dr. Jessica Bloom (Children’s Hospital Colorado), Dr. Marta Moura (Mayo Clinic), Dr. Kinanah Yaseen (Cleveland Clinic) and Dr. Divi Cornec (Brest University in France).

Expand your knowledge of vasculitis. Visit the Vasculitis Foundation website at: https://vasculitisfoundation.org/

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Vasculitis Patient-Powered Research Network VPPRN Update
Recorded July 2023

Dr. Merkel discusses the VPPRN’s mission and successes, patient data and engagement, membership, and the primary entities that collaborate with the VPPRN. He looks at recent, current, and future VPPRN studies and reviews the VPPRN’s publications. Dr. Gordon follows with the reasons why someone should join the VPPRN and ways to participate in research.

Expand your knowledge of vasculitis. Visit the Vasculitis Foundation website at: https://vasculitisfoundation.org/

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New Medications
Presenter: Dr. Nicole Orzechowski
Chapel Hill 2024 Regional Conference

0:00
1:26 Avocopan
2:33 ADVOCATE: Phase 3 Clinical Trial
5:46 Kidney recovery with Avocopan
9:53 Obinutuzumab
13:00 EGPA
13:48 Mepolizumab
14:36 The MANDARA Study
18:05 CAR-T Cell Therapy
19:41 Summary

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0:00
0:40 Introduction of Dr. Orzechowski

1:48 Would the doctor please go into more detail about the TAVENEOS® (avacopan) trial?

4:45 Is it possible that avocopan could replace steroids, or is the goal to have a drug that
reduces the amount of steroid therapy?

5:30 Is part of the goal with avocopan to lead to remission more quickly?

7:12 In her summary slide, Dr. Orzechowski wrote that the most severe cases of ANCA vasc were
not enrolled in the ADVOCATE study. What are the reasons for this issue? Are there plans to
include these more severe cases.

8:59 How many phases of the ADVOCATE study will there be?

10:50 I’m a patient with EGPA (for six years). I’ve had two relapses. Could the doctor explain (to a
very non-technical person) what the MANDARA study could mean to me. Is she saying this
drug Benralizaumb increases the chances of remission for EGPA patients.

13:26 I’d like to know the process of why some drugs are Infusions and some are injectables. In
general, do they all start as infusions and then the efficacy of making it injectable also has to
be studied?

16:49 Would you comment on the timeline and evolution of these therapies? We hear about
these exciting therapies, but we forget it takes a long time before it’s in the marketplace.

19:34 The role of the VPPRN in research.

Recorded April 2024

Note: Watch Dr. Orzechowski's full webinar on New Meds here:https://youtu.be/SDixlOzN47o

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What are clinical trials and why are they important and how can patients participate?

Presented by: Allyson Egan, BSc(Hons),MRCP, Consultant Nephrologist | Trinity Health Kidney Centre, Tallaght University Hospital, Dublin

The 2024 International Patient Conference Barcelona, Spain, April 6, 2024

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Patient Registries: FairVas

Presented by: Julie Power, Vasculi􀀁s Ireland

The 2024 International Patient Conference Barcelona, Spain, April 6, 2024

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Patient Registries: REVAS

Presented by: Roser Solans-Laque, MD, PhD

The 2024 International Patient Conference Barcelona, Spain, April 6, 2024

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0:00

1:10 Introducing Dr. Free

2:29 Your presentation had a new abbreviation, LTROT. My understanding is this is a goal of
getting the patient is totally off treatment in a state of remission. Is this correct?

5:21: Dr. Free, can you talk about what is meant by immunological remission? Is this the same
idea as achieving any type of remission from vasculitis, or is this a specific type of
remission? Thank you.

7:55 Could you give a layperson explanation of dendritic cells and their purpose? What is the
important takeaway from this process as it pertains to vasculitis?

11:46 You talk about the ultimate goal of restoration of immune system intolerance. A resetting or
redirecting of the immune system. At first, I thought this was the same as finding a cure
where the immune system is restored. Then it seemed like it’s not what we think of as a
cure per se, but to achieve remission. But I’m still confused. Can you elaborate, please?

14:35 Suddenly I’m hearing a lot about CAR and CAAR-T cells. Several doctors have said it’s too
early, too premature …but they didn’t say too premature for what exactly. Do they mean this
research is too premature to provide possible therapeutic options?

19:36 We are hearing about all of the potential, promising therapies. Could you doctor address how
and why it takes so long for these therapies to become approved, new treatments for
patients.

Recorded April 2024

Watch Dr. Free's entire presentation, Emerging Therapies, from the VF Chapel Hill Conference: https://youtu.be/NjAzb6lcVW4

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Exciting Research Advances on the Horizon_Barcelona 2024
Presented by: Peter A. Merkel, MD, MPH, and Richard Kitching, MD, PhD

The 2024 International Patient Conference
Barcelona, Spain
April 6, 2024

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0:00
2:47 The back story about this study on Vitamin D and vasculitis.
8:49 What is Vitamin D?
9:41 Effects of Vitamin D deficiency?
11:23 How much is right for you?
12:56 Research on the benefits of supplementing Vitamin D.
14:47 The Vitamin D controversy.
17:03 Exploring the Vitamin D status in ANCA-Associated Vasculitis.
24:34 Q&A: How was your research presentation received at the 2024 Barcelona Workshop?
31:55 Q&A: How much Vitamin D should we be taking?
33:58 Q&A: Should I be taking Vitamin D if I have active EGPA?
35:46 Q&A: Is there a genetic link to Vitamin D deficiency?
37:01 Q&A: How did this study about the benefits of Vitamin D originate?
40:13 Q&A: Is absorption of Vitamin D affected if a patient has GI involvement with their vasculitis?
41:33 Q&A: What type of Vitamin D should I buy?
44:01 Q&A: Any research to share about the use of Vitamin D to offset the effects of COVID-19?
45:50 Q&A: What are the outcomes that you would like to see from this research?
48:46 Q&A: What are the next steps for this study?

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0:00
2:25 Project background: Steroid reduction.
4:36 Project objectives: Improve safety of treatment of AAV.
5:19 Provide alternative options for GC-sparing regimens.
6:01 Methods and steps.
8:57 Potential benefits of this study.
10:48 Support for this project.
12:11 What is meant by induction therapy? When a doctor first diagnoses a patient how does he/she determine the dosage of steroids needed to be most effective?
16:03 Is one of the goals where newly diagnosed patients could start on a much lower dose of steroids but still achieve control over the inflammation?
19:56 Does the doctor imagine a day when steroids will be eliminated and replaced with another type of therapy (medication) that will be just as effective as steroids, but without the side effects?
25:20 Steroid therapy—how low can we go?
26:07 Is the SAFE-T Study related to the PEXIVAS Trial?
29:23 Are you getting closer to the phase where you will be recruiting patients?
30:58 How did you become interested in nephrology?

The SAFE-T Study: How low can we go with steroid therapy?
August 2024

Addendum: Read a VF article about David Massicotte-Azarniouch
and his current study at: https://tinyurl.com/2sdrxcfb

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0:00
2:34 Audience poll on neurological issues with vasculitis.
9:01 Systemic vasculitis vs. Nonsystemic vasculitis.
12:00 An illustration of our nervous system.
16:03 Imaging: Chronic Peripheral Nerve Vasculitis.
16:50 Symptoms with neuropathy and vasculitis.
18:29 When to suspect vasculitic neuropathy.
19:58 Why does early diagnosis matter?
20:43 Investigations: Clinical examinations.
22:10 Nerve conduction studies /EMG
23:47 Lab testing.
24:33 Nerve biopsy.
26:21 Treatment strategies.
31:48 Summary.
33:14 Q&A: What is it like to get a nerve conduction study?
36:16 Q&A: Can numbness in limbs be a sign of neuropathy?
38:33 Q&A: I have LVV Giant Cell Arteritis but I have been dealing with burning toes (not big toes just small) and also my ankles ache.
39:43 Q&A: Can prednisone mask mobility issues, and then show up later after being weaned off of prednisone?
41:06 Q&A: Is it unusual for a patient whose MPA, as far as serious kidney damage is concerned, is in check by rituximab but who has confirmed asymmetrical nerve damage and resulting neuropathy of hands and feet to have continued worsening of the neuropathy?
43:08 Q&A: What should be done if exercise or massage of the affected area worsens the symptoms of tingling, burning, etc.?
44:01 Q&A: How to find a neurologist who works with vasculitis and sees me as the whole person rather than only a certain part?
45:22 Q&A: I have ANCA vasculitis and had a drop foot in both of my feet two years ago. Now I only have neuropathy in one foot. I don't take any medicine was on gabapentin two years ago. My question is can it go away or does it get worse as we age?
48:14 Q&A: Why does neurotherapy make your feet and legs cold?
49:21 Q&A: Should a patient be referred to physical therapy when he/she has neuropathy?
49:51 Q&A I struggle with neuropathy in my hands and feet but have recently been diagnosed with the same issue in my rib cage. Is this rare?
50:32 Q&A I've been told that 600 mg of Alpha Lipoic Acid daily helps. Do you agree? Are there any supplements that you recommend?
51:40 Q&A Is Raynaud’s considered neuropathy?
52:00 Q&A How is it possible for a patient with a historical diagnosis of carpal tunnel to know if tingling is vascular neuropathy?
53:18 Q&A Does a foot drop from peripheral neuropathy due to vasculitis require wearing a splint for life?
54:39 Q&A Is an ongoing relationship with a neurologist necessary if you have been diagnosed with neuropathy?

Neurologic Manifestations of Vasculitis
Recorded January 2025

View the slide presentation: https://tinyurl.com/ycy4b333

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0:00
1:34 Remarkable changes in the treatment of GPA.
2:34 The role of Rituximab.
4:57 In your practice what markers do you look at to make your decisions?
8:23 Putting out the fire of inflammation. But what about the “embers”?
9:18 Induction and maintenance therapy.
11:15 The issue of not having GPA come back again.
13:10 Managing sub-glottic and tracheal stenosis when flaring.
17:12 Clinical features of ANCA-associated vasculitis.
18:44 GPA and MPA
19:06 Subclassifying AAV: clinical and prognostic information
20:32 Treatment of ANCA-associated vasculitis
21:55 Could you comment on the use of bactrim in the treatment of subglottic stenosis?
25:00 Explaining Bactrim.
27:29 Is there any updated information on Avocopan and combining it with prednisone, or rituximab?
31:06 Avocopan has been well-studied with ANCA-positive patients. Can you comment on testing Avocopan with ANCA-negative vasculitis?
39:11 What are the long-term effects of rituximab on young adults?
42:17 Research into possible treatment for people allergic to rituximab.
42:46 Looking into CAR T-cell therapy.
44:06 What do you think of a chronic low dose of prednisone?
52:44 What are some early symptoms of sub-glottic stenosis?

Granulomatosis with Polyangiitis
Recorded July 2023

In a Q&A format, Drs. Merkel, Berti, and Stein answer general questions from the audience about GPA, treatments, new ideas in research, individual manifestations and the progress that has been made over the years.  Peter A. Merkel, MD, MPH University of Pennsylvania.

Expand your knowledge of vasculitis. Visit the Vasculitis Foundation website at: https://vasculitisfoundation.org/

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0:00
5:11 Fun facts about the microbiome
6:39 What does your gut microbiome do?
8:30 Microbiome connections
15:15 Diet and the microbiome
15:49 Microbiome and autoimmunity
16:51 Microbiome and vasculitis
18:21 Inflammation of the nose and sinuses
21:02 Unanswered questions
21:45 A unique longitudinal biorepository focused on sinonasal disease
24:00 Focusing on what happens before relapse (pre-relapse)
24:52 Do nasal bacteria change prior to and during relapse of GPA?
26:15 Corynebacteria: an understudied bug
28:42 Molecular changes occurring in nasal tissue observed prior to relapse
31:13 Nasal cells of GPA are different from those of healthy patients
34:00 Long-term goal: unravel the relationships between microbes, sinonasal cells, and the immune system
35:14 Vasculitis Sino-Nasal Indicators For Flares (V-SNIFF) Study
37:07 Misconceptions about the microbiome
40:00 Risks of probiotics
43:31 Q&A

The Microbiome and Vasculitis
March 2025

View the slides from this presentation: https://vasculitisfoundation.org/wp-content/uploads/2025/03/Microbiome_VF_Rhee2025.pdf

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CAR-T cell therapy is one of the most exciting frontiers in autoimmune disease research—and it may hold promise for the future of vasculitis treatment. In this VF webinar, Dr. Meghan Free and Dr. Dhruti Chen from the University of North Carolina for an engaging, patient-friendly webinar that explains this cutting-edge therapy in simple terms.

0:00
3:35 Blood Cell Primer
4:39 Lymphocytes
5:38 What is CAR-T
8:30 CAR-T cells approved for cancer
8:52 Evolution of CAR-T cells
9:44 Why could CAR-T therapy help vasculitis?
10:44 CD19 – CAR T cells in autoimmune disease
12 :42 What results have been seen so far?
13:40 Specific trials to note, including vasculitis
16:41 Research is still preliminary
17:40 Risks and practical considerations (patient view)
19:45 Why patients should be aware (key takeaways)
21:42 Pros and cons
24:36 Practical next steps and resources
24:39 How do you define refractory vasculitis?
26:48 Q: Is CAR T therapy expected to require repeated treatments at regular intervals? After the body replenishes B cells?
27:50 Q: It’s a promising therapy, but you can talk about why expectations need to be tempered with where we are right now.
29:03 Q: Just a rough guesstimate,e how long do you think it could be until it would be a standard of care?
30:15 Q: I have a daughter with hypocomplementemic urticarial vasculitis syndrome. If CAR-T cell therapy could potentially put her in remission, why would it make more sense to keep her on mycophenolate? She is six years old and has been on Myco since June 2024
31:14 Could you tell us about the efficiency and toxicity of CAR-T compared to Rituximab?
33:12 You have referred to ANCA Vasculitis. What about non-ANCA Vasculitis?
34:00 Can you explain CAR-T cells vs CAR-NK cells?
35:26 Could the doctors explain about genetically modifying a cell? Could you speak to that idea to help reduce the concern?
37:28 Could the doctors talk about lympo depletion, that also sounds concerning.

Visit the VF website to discover all of our resources on vasculitis. www.vasculitisfoundation.org

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