4 Hematology Headlines You Missed in March 2026


Recent updates in hematology underscore simultaneous progress in therapeutic innovation, precision diagnostics, and efforts to standardize care across rare and complex blood disorders.

In polycythemia vera (PV), the US Food and Drug Administration (FDA) has granted Priority Review to rusfertide, a first-in-class hepcidin mimetic targeting iron dysregulation, based on phase 3 VERIFY and supportive long-term data demonstrating improved hematocrit control, reduced phlebotomy requirements, and symptom relief versus standard of care.

In parallel, the American Society of Hematology (ASH) has released updated guidelines for severe and very severe acquired aplastic anemia, outlining 33 recommendations and 4 good practice statements. The guidance emphasizes earlier use of genetic and diagnostic testing, individualized treatment strategies, including immunosuppressive therapy with eltrombopag, and clearer pathways to second-line care to improve outcomes in this high-risk population.

Advances in molecular diagnostics are also reshaping hematologic malignancy care, with rapid next-generation sequencing (NGS) enabling faster identification of actionable mutations to guide early treatment decisions. However, uneven access, variability in reporting, and infrastructure limitations continue to constrain real-world implementation, highlighting a persistent gap between technological capability and clinical deployment.

At the same time, workforce data highlight ongoing system-level strain, with hematology-oncology physicians treating sickle cell disease reporting higher burnout rates than peers despite similar levels of resilience. Together, these developments reflect a field advancing rapidly in scientific capability while still facing significant challenges in equitable access, implementation, and provider sustainability.

Catch up on the 4 headlines you missed from March 2026:

FDA Accepts New Drug Application for Rusfertide, Grants Priority Review In Polycythemia Vera

The FDA has accepted the New Drug Application for rusfertide and granted Priority Review, positioning the investigational therapy as a potential first-in-class option for polycythemia vera (PV). Based on phase 3 VERIFY and supportive long-term data, rusfertide demonstrated improved hematocrit control, reduced phlebotomy needs, and symptom benefits compared with standard care alone.

The hepcidin mimetic, which targets iron dysregulation underlying PV, was generally well tolerated, with mostly low-grade adverse events reported. A PDUFA decision is expected in the third quarter of 2026.

Burnout in Sickle Cell Physicians Hits 60% While Grit and Resilience Stand Strong

Burnout rates were higher among hematology-oncology physicians treating sickle cell disease (60%) versus those who do not (43%), despite similar levels of grit and resilience. Survey findings suggest systemic factors, rather than individual behaviors, are driving the disparity, with lower job pride, reduced recreation time, and compensation differences contributing to increased burnout.

Timing Is Everything: Advancing Genomic Testing in Hematologic Malignancies

Rapid next-generation sequencing (NGS) is improving precision care in hematologic malignancies by enabling timely identification of actionable mutations to guide early treatment decisions. However, adoption remains uneven, with barriers including limited expertise, inconsistent reporting, and variability in access and reimbursement across health systems.

ASH Publishes Guidelines on Diagnosis and Management of Severe and Very Severe Acquired Aplastic Anemia

ASH has released updated guidelines outlining 33 recommendations and 4 good practice statements for the diagnosis and management of severe and very severe acquired aplastic anemia.

The guidance emphasizes improved diagnostic strategies, including expanded genetic testing, and supports personalized treatment approaches such as early use of eltrombopag alongside immunosuppressive therapy and timely transition to second-line options. Aimed at improving outcomes in this rare but life-threatening condition, the recommendations also seek to expand access to appropriate therapies and standardize care across patient populations.

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Catch up on the hematology headlines you missed in March 2026. 

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4 Hematology Headlines You Missed in March 2026

Ryan Livingston joined the MJH Life Sciences team in March of 2025, shortly after graduating from TCNJ with a bachelor’s degree in English. He enjoys creative writing, reading, and scuba diving. He can be reached at rlivingston@mjhlifesciences.com.

On April 6, 2026, Amgen announced positive topline results from a phase 3 trial of teprotumumab-trbw (Tepezza), administered via an on-body injector (OBI), for the treatment of moderate-to-severe 

Tepezza was first approved by the US Food and Drug Administration in 2020 and is the first and only approved medicine for TED. It is designed to reduce proptosis and double vision – 2 hallmark symptoms of TED – and has demonstrated its efficacy across several global clinical studies. Additionally, the drug’s safety and efficacy are well-established via 6 years of real-world experience in >25,000 patients.

“These results extend and support the best-in-class efficacy of Tepezza for people living with thyroid eye disease, now with subcutaneous administration delivering IV-level efficacy,” Jay Bradner, MD, executive vice president of research and development at Amgen, said in a statement. “With a well-understood mechanism and established impact in the clinic, we can evolve how the medicine is delivered to potentially reach even more patients through a more convenient subcutaneous option.”

The present study was a randomized, double-masked, placebo-controlled, parallel-group, multicenter trial, aiming to evaluate the efficacy and safety of subcutaneously administered Tepezza in patients with active TED compared to placebo. Investigators included a primary endpoint of proptosis responder rate, measured as the percentage of participants with a ≥2mm reduction from baseline in the study eye without deterioration of proptosis in the fellow eye by week 24.

To be eligible for inclusion, patients had to have a diagnosis of moderate-to-severe active TED within 15 months and proptosis of ≥3 mm from baseline prior to TED diagnosis, among other criteria. Patients were administered Tepezza or placebo via an OBI every 2 weeks, totaling to 12 injections.

Ultimately, the trial met its primary endpoint, reflecting a statistically significant and clinically meaningful 77% proptosis response rate (76.7% Tepezza OBI vs 19.6% placebo; 

 <.0001). Additionally, the mean proptosis reduction was -3.17 mm by week 24 (-3.17mm Tepezza OBI vs -0.8 mm placebo; 

Investigators also saw significant improvements in overall responder rate, the percentage of patients achieving a Clinical Activity Score (CAS) of 0 or 1, change in diplopia as ordinal response categories, diplopia response rate, and mean change from baseline in week 24 in the Graves’ Ophthalmology Quality of Life (GO-QoL) appearance subscale, among other secondary endpoints. Despite not achieving statistical significance, the team saw a numerical trend in the mean change in baseline at week 24 in the GO-QoL visual functioning subscale favoring Tepezza OBI.

“Thyroid eye disease can be a profoundly debilitating condition, affecting not only vision but also daily functioning with symptoms like double vision and eye bulging,” Madhura Tamhankar, MD, professor of ophthalmology and neurology at the Scheie Eye Institute, University of Pennsylvania, said in a statement. “Expanding administration options through subcutaneous delivery opens the possibility of a more accessible experience for patients with thyroid eye disease and is critical to serving diverse patient needs. The potential to achieve comparable efficacy to IV makes this advancement compelling.”

Amgen. Amgen Announces Positive Topline Phase 3 Results for Subcutaneous Tepezza in Adults Living With Moderate-to-Severe Active Thyroid Eye Disease. April 6, 2026. Accessed April 6, 2026. 

https://investors.amgen.com/news-releases/news-release-details/amgen-announces-positive-topline-phase-3-results-subcutaneous

US Food and Drug Administration. FDA approves first treatment for thyroid eye disease. 

. January 21, 2020. Accessed April 6, 2026. 

https://www.prnewswire.com/news-releases/fda-approves-first-treatment-for-thyroid-eye-disease-300990621.html

Amgen reported positive phase 3 topline data for teprotumumab-trbw delivered subcutaneously via an on-body injector in moderate-to-severe active thyroid eye disease. In a randomized, double-masked, placebo-controlled trial, dosing every two weeks for 24 weeks achieved a 77% proptosis responder rate versus 19.6% with placebo and a mean proptosis reduction of 3.17 mm. Secondary endpoints improved across overall response, CAS, diplopia metrics, and GO-QoL appearance; visual functioning trended favorably. Subcutaneous delivery may broaden access while maintaining IV-like efficacy.

Amgen has announced positive topline results from their phase 3 trial comparing teprotumumab-trbw – administered via an on-body injector – to placebo.

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Tepezza OBI Achieves Proptosis Reduction in Thyroid Eye Disease in Phase 3 Trial

Jane M. Grant-Kels, MD, is the vice chair of the Department of Dermatology and founding director of the Cutaneous Oncology Center and Melanoma Programs and a professor of dermatology, pathology, and pediatrics at the University of Connecticut Health Center.

A presentation titled ‘Making the Most of Health Information Technology: Tools for the Dermatology Practice’ was given at the 

2026 American Academy of Dermatology (AAD) Annual Meeting

Jane M. Grant-Kels, MD, was the session presenter, and she was interviewed with HCPLive about key takeaways on the topics highlighted in ths talk. Grant-Kels is known for her role as the vice chair of the Department of Dermatology, the founding director of the Cutaneous Oncology Center and Melanoma Programs, and as a professor of dermatology, pathology, and pediatrics at the University of Connecticut Health Center.

 interview Q&A features Grant-Kels discussing notable highlights from her talk at AAD 2026:

 We want to ask about your other AAD session on making the most of health information technology tools for dermatology practice. Would you tell us about some of the tools or practices that you highlight within that session?

A lot of the new tools involve artificial intelligence. Artificial Intelligence is now being used to help streamline paperwork, fill out forms, communicate with patients on the website, and is also now being used for scribe work. So very often, you'll go into a room with a new doctor or a doctor, and they'll say, ‘Do you mind if I record this session?’ Because the recording is what's required for the AI to create a note out of the conversation that you're having…Those are great things. I'm a big advocate for doctors not being afraid of AI, but embracing it and making their practices more efficient. From the patient's perspective, there are concerns, though. First, a lot of patients are afraid of artificial intelligence and that you need to get their informed consent for you to use it, particularly for an AI scribe.

Then the other big issue is privacy, because how that data is saved is complex, and as we all know, there is no such thing as 100% secure data anymore, and AI only makes that more of a difficulty, because that data, especially a visit with the doctor, where all the information is transcribed, is no longer as secure as it was when we had written records. The electronic medical record, per se, makes data available to others, but it can also be broken into. So those are the main, main issues. The other issue is access to something called distributive justice. AI is very expensive and to include and who is going to have the ability to have that part of their practice, but are going to be physicians who are practicing in more affluent areas, rather than physicians who are taking care of Medicaid patients or rural doctors who may not have access to a higher population that can pay more, so that you know the availability of all this new technology is also not going to be distributed fairly because of the cost involved.

Those are the major issues. Informed consent, autonomy, the patient's right to allow or not allow AI to be part of the visit, and distributive justice. Who's going to have access to all of these new technologies that are very expensive? Those are the major issues. Then, there's also the concern that AI is going to replace healthcare providers in some form or fashion, whether it's the physicians themselves, whether you will need fewer physicians, or it's the secretary at the front desk or the administrator who does the paperwork.

The question is, how do we embrace AI incorporated into our practices and ensure that people have job training so that they know how to use the AI and can be employed in some fashion, to assist in the healthcare system? So those are the major concerns that I'm going to bring up about the employment of the new technologies…For anybody who's called a number like you call a department store or business, and you're never talking to a human being anymore, that's beginning to happen in medicine now. You call on someone's office, and you're not talking to an answering service. You're not talking to their front desk service.

Very often, you're talking to an AI that sounds very human-like and may be answering your questions. And you know, there's a concern about accuracy, if they're deliberating with the patient, and even in some cases, giving advice to the patient. A lot of these AI services, where the patient has access to their electronic medical record and can ask questions, in many cases. Now, the initial questions may be handled by AI, and there's some concern about how accurate that is as well.

Have you noticed in particular that there is one form or another of this technology that is more widely implemented in your own practice or any other notable products you highlight in your session?

There's been an explosion of products that are available. I mean, all you have to do is type in AI and medical, you know, office care, and it explodes with different companies that you know, there are many, many companies, and I applaud them trying to streamline prior authorization. So when you come to me as a patient, and I write a prescription, the insurance company and their wisdom turns it down, and then you have to fill out a prior authorization to rationalize why you want that drug.

This is very time-consuming for offices, and so many systems now offer AI to prepare the paperwork. Of course, at the same time, the insurer is also employing AI to reject the new prior authorization that you've submitted. But there's been an explosion. The main electronic health record that a lot of people are using in hospitals is, obviously, Epic. Epic is now employing AI in some of its technology. So that's what I'm exposed to, mostly at my institution. But in the private sector, people can use whatever they want.

Do you feel there is a concern, maybe among medical residents, about AI scribes and AI note takers removing the ability to learn certain things as thoroughly?

A lot of people, historically, have worked as scribes before medical school. That's when they finished college. They're either taking some pre-med, and they're working part-time, ascribed, learning the lingo, learning the medical, electronic medical record, and those jobs are definitely at risk, and I think a lot of people who have enhanced people's education and prepared them better for medical school. There is that concern. I think, from my experience, I find residents and medical students worried about what their role will be and whether they're going to be replaced with AI, for example, in radiology. Most X-rays are read by AI and then reviewed by a radiologist in pathology.

Now, a lot of slides are reviewed by AI and then checked by a pathologist…Dermoscopic images can now be evaluated by AI. I teach courses on demoscopy, and I'm also a dermatopathologist, and residents ask, ‘Do I have to learn this if AI is going to do it for me?’ The answer is yes, because AI needs the human as well as the AI together to be a partner, because it sometimes makes mistakes, just like we as humans make mistakes. The other problem with a lot of the AI is [one that is] in dermatology. A lot of the training sets have not included all skin types, and so there's a big concern whether the AI is more prone to make mistakes with darker skin patients, because it hasn't been trained properly.

So there are a lot of concerns about the accuracy of AI, the privacy, the informed consent, the autonomy of the patient, and the role that we as physicians will have with AI as it gets better, because AI tends to get better and better. So you know, there's this always and it's learning from its mistakes. So the question is, will it someday? Will there not be a need for dermatopathologists? Will there not be a need for demoscapists? Will there not be a need for radiologists? And my answer is yes, there will be a need. It will enhance our job by circling an area with its notes on pathology. But that doesn't mean we don't look at the slide, or that we don't look at the X-ray, but there is that concern among younger physicians today.

Can you envision a world in which these new technologies enhance the jobs of some of these clinicians, so they will actually be better able to tailor things specifically toward patients a little bit more, rather than the current system?

 Physicians are in a very bad situation. Our overhead is going up, just like everybody else's, and our reimbursements have gone down. And so to keep a practice viable, physicians have been forced to see more patients. It's, believe me, doctors don't enjoy going at that speed. I do think AI might help, because it might certainly, in theory, if AI writes the note, that's more time I can look at the patient rather than staring at the computer and typing. I mean, that's the goal. If AI can synthesize and give me a synopsis of what previously happened at previous visits and give me a summary paragraph, I can go into a room more alert to what the patient's concerns are.

I mean, that is possible. But there's a limit to how fast doctors can go, because there's still that human need to come into contact with a patient, and put hands on the patient in regards to reassurance and explaining things to the patient. I do think that for AI, we need to partner with it. I also think that the system needs to change. Reimbursements need to rise with overhead, so that needs to be addressed as well, especially since AI is very expensive. These technologies are not cheap. They come at a great cost to the physician's offices. So there needs to be a compromise, because you can't keep going faster and faster and still get any.

The quotes contained in this discussion were edited for the purposes of clarity.

Grant-Kels J, et al. F062 Making the Most of Health Information Technology: Tools for the Dermatology Practice. Sesson presented at: 2026 American Academy of Dermatology Annual Meeting; March 27–31, 2026; Denver, CO.

AI adoption in dermatology is accelerating, especially for documentation, patient messaging, and administrative automation such as prior authorizations. Benefits center on reducing clerical burden and improving visit efficiency, potentially returning clinician attention to the patient rather than the EHR. Major implementation barriers include informed consent for AI scribes, privacy and cybersecurity risks, and equity concerns because high costs may concentrate tools in affluent practices. Additional tensions involve AI-mediated patient communication accuracy, workforce displacement, and training implications, alongside bias from non-representative dermatology image datasets.

This Q&A interview at AAD 2026 highlights health information technologies and AI tools used in dermatology practices.

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Tips for AI and Health Information Technology in Dermatology Practice, With Jane M. Grant-Kels, MD

Linda Stein Gold, MD, is a dermatologist at Henry Ford Health. 

Eingun James Song, MD, is the director of clinical research for Frontier Dermatology. 

Value of Utilizing Matching-Adjusted Indirect Comparisons (MAICs) in Psoriasis

In this episode, "Value of Utilizing Matching-Adjusted Indirect Comparisons (MAICs) in Psoriasis," the expert dermatologists explore the methodology behind matching adjusted indirect comparisons and their practical value in guiding evidence based treatment decisions for plaque psoriasis.

The panel opens by establishing that while head to head clinical trials remain the gold standard for comparing therapies, they are not always available. In their absence, matching adjusted indirect comparisons offer a more statistically rigorous alternative to simply comparing results across separate trials. The methodology is explained clearly: when patient level data is available for one drug and only aggregate mean demographic data is available for another, the patient level dataset can be adjusted and matched to the population characteristics of the comparator drug. This process accounts for known variables that can influence treatment outcomes, such as prior biologic exposure, disease severity, and the prevalence of psoriatic arthritis in the study population, resulting in a more balanced and meaningful comparison between the two therapies.

The panel emphasizes that matching adjusted indirect comparisons are not only valuable to manufacturers seeking to demonstrate their drug's performance to payers, but also serve as an important clinical decision making tool for dermatologists navigating treatment sequencing without direct comparative data. The discussion concludes with the panel reinforcing that patients who receive the most effective drug first tend to achieve better long term outcomes than those who cycle through multiple therapies, making the ability to identify a preferred agent through indirect comparison models a meaningful advantage in optimizing patient care.

The next episode in this series, "Evaluating Psoriasis Therapies using MAICs," features the panelists advancing their conversation on psoriasis and focusing on recent MAIC analyses comparing key IL-23 inhibitors and other biologic classes, highlighting how these indirect comparisons have demonstrated statistically significant efficacy differences across PASI endpoints and have since been validated by dedicated head to head clinical trials.

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Analyzing IL-23 Inhibitors in Psoriasis

This episode, titled "Analyzing IL-23 Inhibitors in Psoriasis," features panelists discussing the future of IL-23 targeted therapies and the practical considerations surrounding the anticipated shift toward oral treatment options in plaque psoriasis.

The panel opens by surveying the emerging therapeutic pipeline, noting that most innovations continue to target the IL-23 pathway either directly or indirectly, underscoring the cytokine's central role in psoriasis pathogenesis. Key agents discussed include an oral macrocyclic peptide IL-23 receptor blocker, which directly binds the receptor rather than the cytokine and has demonstrated complete skin clearance in approximately half of patients at 24 weeks, a level of efficacy described as unprecedented for an oral therapy and comparable to leading biologics. Two next generation TYK2 inhibitors are also highlighted, offering higher binding affinity and selectivity than existing options and indirectly antagonizing IL-23 signaling with more rapid and deeper responses. Additionally, a monoclonal antibody called ORKA-001 is discussed, notable for its half-life approximately four times greater than risankizumab and its potential for dosing intervals of every 6 to 12 months in responding patients.

From a safety standpoint, the panel notes that IL-23 targeted therapies continue to demonstrate a favorable profile, with only occasional mild adverse events such as upper respiratory tract infections or fungal infections.

The discussion then shifts to the practical implications of oral versus injectable IL-23 therapies in real world settings. The panel acknowledges that while clinical trials demonstrate strong efficacy for emerging oral agents, real world adherence remains an open question, with concerns around daily pill taking, administration requirements such as empty stomach dosing, and potential drug interactions. Injectable biologics are noted as having a relative adherence advantage given their infrequent dosing schedule and easier monitoring. The panel agrees that needle phobia, while real for some patients, affects a smaller population than commonly assumed, particularly when injections are spaced weeks or months apart. Ultimately, the panel expresses enthusiasm for expanded treatment options, emphasizing that real world evidence will be critical in determining how well emerging oral therapies perform outside of controlled trial settings.

In the next episode, "Value of Utilizing Matching-Adjusted Indirect Comparisons (MAICs) in Psoriasis," panelists will continue their discussion on psoriasis and highlight the methodology and clinical value of matching adjusted indirect comparisons as a rigorous statistical tool for comparing biologic therapies in the absence of head to head trial data, and how this evidence can inform treatment sequencing decisions in real world practice.

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The HCPFive: Top News for Healthcare Providers from the Week of 03/29

This Q&A interview with Grant-Kels highlights takeaways from her session on managing the needs of patients in dermatology practicers who are pregnant.

Advice for Evaluating Pregnant Patients in Dermatology, With Jane M. Grant-Kels, MD

Diana Isaacs, PharmD, is an endocrine clinical pharmacist, director of Education and Training in Diabetes Technology, and codirector of Endocrine Disorders in Pregnancy at the Cleveland Clinic.

Podcasts

This episode covers the March 26th approval of Awiqli for T2D, highlighting its high concentration and once-weekly administration.

Type 2 Diabetes

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Diabetes Dialogue: FDA Approves Novo Nordisk’s Insulin Icodec-abae (Awiqli) for T2D

Mardi Gomberg-Maitland, MD, MSc, is the director of the pulmonary hypertension program, chief clinical research officer at the Office of Clinical Research SMHS, and a professor of medicine at George Washington University.

Gomberg-Maitland addresses sotatercept’s capacity to improve pulmonary vascular and cardiac endpoints in patients with CpcPH-HFpEF.

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CADENCE: Sotatercept Reduces PVR in CpcPH-HFpEF, With Mardi Gomberg-Maitland, MD, MSc

Sayna Norouzi, MD, is an assistant professor of medicine and clinical nephrologist at Loma Linda University Medical Center

Norouzi discussed the remaining challenges that come with having evolving treatment options and a robust patient population in IgAN.

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New Therapeutic Options Raise More Questions for IgA Nephropathy Treatment, With Sayna Norouzi, MD

Catch up on the most impactful trial readouts to come out of ACC 2026.

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Cardiomyopathy

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ACC 2026: Phase 2/3 Trials to Know

Tim Smith joined the MJH Life Sciences team in 2022 and is currently an associate editor for HCPLive. He is also the executive producer of two podcasts: Skin of Color Savvy and The Medical Sisterhood. Previously, he was the producer of the Lungcast podcast. He graduated from UC Berkeley and worked in multimedia journalism as a staff writer prior to joining MJH. You can contact him at tsmith@mjhlifesciences.com.

This review of new data on psoriasis treatments from AAD 2026 highlights several key trials.

Plaque Psoriasis

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AAD 2026: 6 Psoriasis Trials to Know

Alex Hillenbrand is an assistant editor for 

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Catch up on the guideline updates, trial news, and featured content from March 2026. 


Chronic Kidney Disease

5 Nephrology Headlines You Missed in March 2026

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Diabetes Dialogue: 2026 AACE Guidelines for Type 2 Diabetes

Victoria Johnson joined MJH Life Sciences in 2020 and has written for NeurologyLive, CGTLive, and is now an assistant managing editor for HCPLive and RheumatologyLive. She doesn’t have enough time for her numerous hobbies. You can reach her at vjohnson@mjhlifesciences.com.

Check out 6 FDA decisions to watch in Q2 2026, listed chronologically by expected decision date.

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4 Hematology Headlines You Missed in March 2026

Tepezza OBI Achieves Proptosis Reduction in Thyroid Eye Disease in Phase 3 Trial

Tips for AI and Health Information Technology in Dermatology Practice, With Jane M. Grant-Kels, MD

Value of Utilizing Matching-Adjusted Indirect Comparisons (MAICs) in Psoriasis

Analyzing IL-23 Inhibitors in Psoriasis