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.

American Heart Association’s Scientific Sessions 2025

, in New Orleans, Louisiana, were – as always - a hotbed for critical cardiology news. Everything from 

atherosclerotic cardiovascular disease (ASCVD)

 saw exciting new data, with plenty of critical trial results announced and upcoming therapies showcased.

Given the sheer amount of news released from November 8-10, 2025, the

 editorial team has put together a list of the 4 most critical trials for clinicians to know from AHA 2025 – check them out below.


DECAF: Caffeine Consumption Does Not Increase, May Improve Atrial Fibrillation Risk

The Does Eliminating Coffee Avoid Fibrillation (DECAF) trial investigated the effect of a single cup of coffee per day on adult patients who had been treated for atrial fibrillation. Investigators noted a 39% decrease in irregular heart rhythm episode risk among patients who drank coffee than those who avoided all caffeine, which may indicate a potentially beneficial effect of caffeine on the heart. However, despite these positive results, investigators still urge caution in interpreting these data, as they have not been adjusted for multiplicity.


CORALreef Lipids: Enlicitide Lowers LDL-C as Much as Injectables, With Ann Marie Navar, MD, PhD

This phase 3 trial compared investigative oral PCSK9 inhibitor enlicitide to placebo in patients with hypercholesterolemia who either had or were at risk for ASCVD, highlighting enlicitide’s superior efficacy in reducing LDL cholesterol. In addition to reducing LDL-C by up to 60%, enlicitide saw a 53% reduction in non-HDL, a 50% reduction in ApoB, and a 28% reduction in Lp(a). The drug’s oral formulation may also make treatment adherence significantly easier compared to existing injection-based therapies.

 editorial team spoke with Ann Marie Navar, MD, PhD, an associate professor at the University of Texas Southwestern Medical Center and lead study author, to discuss the implications of these data.


CRISPR Gene Editing Therapy CTX310 Lowers LDL-C and Triglycerides in Lipid Disorders

CTX310, a one-time CRISPR-Cas9 gene-editing therapy, safely reduced both LDL cholesterol and triglycerides in adults with difficult-to-treat lipid disorders in the first-in-human trial of gene editing targeting ANGPTL3. These unprecedented results, if confirmed in larger-scale trials, could present a one-time treatment for patients with lifelong lipid disorders, significantly reducing the risk of cardiovascular disease. This could revolutionize a disease in which treatment adherence is often the biggest roadblock to lasting cholesterol maintenance.


VESALIUS-CV: Evolocumab Outperforms Placebo in MACE Prevention in ASCVD and Diabetes

The addition of PCSK9 inhibitor evolocumab to existing high-intensity cholesterol-lowering therapy could significantly reduce the risk of a first major adverse cardiovascular event (MACE) in patients with atherosclerotic cardiovascular disease or diabetes. With over 12,000 patients included in the study, investigators found a 25% lower risk of 3-point MACE and a 19% lower risk of 4-point MACE among the patients assigned to evolocumab compared to the placebo arm.

Articles

The American Heart Association's Scientific Sessions 2025 in New Orleans presented significant advancements in cardiology, including trials on dyslipidemia and atherosclerotic cardiovascular disease. The DECAF trial suggested potential benefits of caffeine in reducing atrial fibrillation risk, while the CORALreef Lipids trial demonstrated the efficacy of oral PCSK9 inhibitor enlicitide in lowering LDL cholesterol. CTX310, a CRISPR gene-editing therapy, showed promise in treating lipid disorders. The VESALIUS-CV trial highlighted evolocumab's effectiveness in reducing major adverse cardiovascular events in patients with ASCVD or diabetes.

Data from 4 of the most impactful trials presented at the American Heart Association Scientific Sessions 2025.

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Dyslipidemia

Atherosclerotic Cardiovascular Disease

Conference

 conference coverage page features articles, videos, and expert-led live coverage from major medical meetings throughout the year.

AHA 2025 Conference Recap: 4 Late-Breaking Trials to Know

Discussing New Topical Drugs for Atopic Dermatitis in Younger Patients, With Peter Lio, MD

2025 Fall Clinical Dermatology Conference

 in Las Vegas, featuring Peter A. Lio, MD, a clinical professor of Dermatology at the Icahn School of Medicine at Mount Sinai and clinical assistant professor of Dermatology & Pediatrics at Northwestern University’s Feinberg School of Medicine.

In this interview at the Fall Clinical meeting, Lio discussed highlights from several of the sessions he co-presented during the conference.

First, Lio was asked about his session on the changing landscape of topical therapies in the field of 

“Topical therapy is so important to me, and it's near and dear to my heart,” Lio explained. “...I think that the money that a company can make from a topical is nowhere near what they can make from a systemic. So to me, it's kind of the ultimate honor for us when companies do it. And I'm so happy we have some really exciting new topicals in our armamentarium, particularly in the world of atopic dermatitis. We have a new topical phosphodiesterase 4 inhibitor that's just a couple of years old now.”

This drug, Lio noted, was topical roflumilast, and it received an indication down to age 2 years recently. Lio went on to discuss other topical medications of note.

“We’ve got topical ruxolitinib, and that is, of course, a topical JAK inhibitor,” Lio said. “That also very recently, in the past month or 2 here, has gotten approval down to age 2. We have topical tapinarof, which is the aryl hydrocarbon receptor agonist and is really awesome. Also approved down to age 2. So not only do we have new treatments for atopic dermatitis, but also in little kids, which is incredible. Most recently, we got a new topical in the class for JAK inhibitors, with topical delgocitinib…for chronic hand eczema.”

Lio went on to discuss some of the most notable takeaways from his portion of the conference session titled ‘30 Tips in 30 Minutes.’

“I think number 1 is going to be the writing out of an action plan,” Lio said. “I think this sort of defines my entire practice, [where] I print out for each individual patient exactly what I'd like them to do. That tells them what to do in the morning, what to do in the evening. It has an order of operations. It links to the products. So they really have all of that comprehensive stuff done. Not only is it for the patient, but it's also selfishly for me because if they call later, then sometimes I'm like.”

Lio went on to describe the second tip provided in his talk, which was a strategy for dealing with patients’ needle-phobia and procedure phobia.

“I have a whole bunch of kind of pearls within the pearl of doing everything you can to minimize the pain, the discomfort, and the anxiety around doing procedures from a blood draw to a biopsy to even just freezing a wart,” Lio said. “All of these things can cause a lot of trauma to the patients. And if especially for [kids], of course, we realize that if they are comfortable, if they are relaxed and on board, not only is that good for them…but it's good for us because we're much more likely to have a successful procedure. And there's even data to support that.”

Lio highlighted the benefits of habit reversal therapy, which he described as a way to give patients a ‘replacement behavior.’

“Instead of scratching at itchy skin, we might have them do something like make gentle circles or tap their fingers, or play with ‘worry beads’ or a fidget toy,” Lio explained. “It sounds kind of goofy, but it really can make a huge difference. It's not the whole story. It's not going to replace an oral JAK inhibitors or biologics. I'm not trying to make a claim like that, but it can be a really nice, supportive or adjunctive piece that addresses the mind body. And sometimes it can literally be the difference between treatment success and treatment failure. I need all the help I can get.”

To find out any additional information on these topics and others highlighted in Lio’s sessions, view the video above. For more on new research in dermatology, view the 

The quotes implemented in this interview summary were edited for clarity.

Relevant disclosures for Lio include AbbVie, Almirall, Arbonne, ASLAN Pharmaceuticals, Dermavant, Eli Lilly and Company, Galderma, Johnson & Johnson, L’Oreal, LEO Pharma, Pfizer, Regeneron/Sanofi Genzyme and others.

Stein Gold L, Frey C, Lio P, et al. The Changing Landscape of Topical Therapies. Session presentation at the 2025 Fall Clinical Dermatology Conference, Las Vegas, Nevada, Oct 23-26, 2025.

Elewski B, Alexis A, Lio P, et al. 30 Tips in 30 Minutes. Session presentation at the 2025 Fall Clinical Dermatology Conference, Las Vegas, Nevada, Oct 23-26, 2025.

Lio P. How To Get Involved in Clinical Trials. Session presentation at the 2025 Fall Clinical Dermatology Conference, Las Vegas, Nevada, Oct 23-26, 2025.

Videos

This Fall Clinical Dermatology Conference interview features Peter Lio, MD, discussing takeaways from several of his sessions at the meeting.

Atopic Dermatitis

Atopic Dermatitis condition center page, audiences can find a variety of coverage mediums for medical news and updates, including articles, video interviews, podcasts, and breaking news on dermatitis research, treatment, and drug development.

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Emily Lau, MD, is a professor of cardiology at Harvard University

Broadening Access to Cardiovascular Care for Female Patients, With Emily Lau, MD

Virtual shared medical appointments (SMAs) are a feasible solution for closing care gaps for female patients with elevated cardiometabolic risk and/or established 

Cardiovascular disease is the leading cause of death among women in the US. An increasing amount of evidence regarding sex- and gender-based differences in cardiovascular disease outcomes has indicated the necessity of more efficient approaches to screening.

 in New Orleans, Louisiana, by Emily Lau, MD, cardiologist at Massachusetts General Hospital and Assistant Professor of Medicine at Harvard Medical School, and colleagues. The team collaborated with Systole Health, a group virtual care organization, to design the study.

 sat down with Lau to discuss the importance of enhancing access to cardiovascular care for women. Lau discussed the factors she sees as driving these treatment gaps.

“I think it’s largely due to a number of reasons. I think that, as physicians, we often see an implicit bias to be less aggressive with our prevention goals in female patients,” Lau told 

. “There is also an access issue; many of our female patients are caregivers themselves, and what is often least prioritized is their own health. And finally, I think there’s a public health education messaging issue, which is to say that despite decades of research demonstrating the opposite, women still think of heart disease as a man’s disease.”

Systole Health enrolled 13 individual cohorts, each consisting of 3 to 5 women, into a 6-week cardiovascular prevention program across 16 US states. Patients were eligible for inclusion if they had documented or self-reported overweight or obesity, hypertension, dyslipidemia, prediabetes, and/or established cardiovascular disease. Patients were excluded if they had unstable cardiovascular disease or were pregnant.

The program included weekly physician-led SMAs, lifestyle health coaching, and peer support. A subset of patients was offered medication management, depending on clinical need. Investigators assessed outcomes, which included self-reported body mass index (BMI), blood pressure, and LDL-C, at baseline and at completion of the program.

Ultimately, 45 women enrolled, collectively contributing 247 patient visits. The mean age across all cohorts was 52 +/- 12 years, and 87% of the population was White. Mean systolic blood pressure at baseline was 124 +/- 15 mmHg, diastolic blood pressure was 78 +/- 11 mmHg, LDL-C was 117 +/- 38 mg/dL, and BMI was 33.4 +/- 7.5 kg/m2. The majority of patients had ≥3 unoptimized cardiovascular risk factors (blood pressure ≥130/80, LDL-C above guideline-recommended thresholds, BMI ≥27, and/or A1c ≥5.7%).

Patients with complete data contributed to a mean change in LDL-C of -35.4% (

 = .001), systolic blood pressure of -7% (

 < .001), diastolic blood pressure of -3% (

 <.001). Investigators also noted that, among patients receiving medication management (N = 20), 36 titrations were performed (mean 1.8 +/- 1.5 per patient) with a median intensification time of 8 days.

This alternative and simplified method of accessing critical features of care for cardiovascular disease may enable clinicians to close care gaps with female patients. Lau also spoke on the value of multiple care models.

“I’ll say this as a cardiologist: we really have an access issue,” Lau said. “There are many more patients who need to see us than we can possibly see. We really need to be thinking about different care models. The traditional care model of people coming in for one-on-one visit into the office or hospital is not meeting their expectations or needs.”

Parmar S, Lee S, Lau E. Closing Prevention Gaps in Women’s Heart Health: Real-World Impact of a Scalable Group Virtual Care Model. Presented at the American Heart Association’s Scientific Sessions 2025. New Orleans, Louisiana. November 8-10, 2025.

Adedinsewo DA, Pollak AW, Phillips SD, et al. Cardiovascular Disease Screening in Women: Leveraging Artificial Intelligence and Digital Tools. Circ Res. 2022;130(4):673-690. 

Lau discusses the use of virtual shared medical appointments for increasing access to cardiovascular care in a traditionally underserved population.

Victoria Johnson joined the MJH Life Sciences in 2020 and has written for NeurologyLive, CGTLive, and now HCPLive. You can reach her at vjohnson@mjhlifesciences.com

American College of Rheumatology (ACR) Convergence

 is widely regarded as a premier annual gathering for rheumatology, showcasing cutting-edge research, clinical advances, and discussions that can shape treatment strategies and patient care.

ACR Convergence 2025, held in Chicago, Illinois from October 25–29 and covered by 

 Rheumatology, featured two late-breaking sessions presenting 24 studies across a range of rheumatologic conditions.

 Rheumatology editorial team has curated 13 of these late-breaking trials, highlighting key findings clinicians should know from the meeting.

Telitacicept Improves Sjögren Symptoms Over Placebo in Phase 3 Trial

Telitacicept, a dual BAFF/APRIL inhibitor, improved systemic disease activity and patient-reported symptoms in Sjögren disease through 48 weeks. 

At Week 24, ESSDAI scores fell by −4.4 with 160 mg and −3.0 with 80 mg versus −0.6 on placebo, and these improvements remained robust through Week 48. By Week 48, 73% (160 mg) and 49% (80 mg) achieved ≥3-point ESSDAI reductions compared with 16.5% on placebo, and ESSPRI improved by −2.56 and −1.74 versus −0.41.

NEPTUNUS-1 and 2: Monthly Ianalumab Significantly Improves Sjögren Disease Activity

Ianalumab, an anti–BAFF-R monoclonal antibody, demonstrated significant improvements in disease activity in patients with Sjögren disease (SjD) in the phase 3 NEPTUNUS-1 and NEPTUNUS-2 studies. 

In pooled monthly dosing data, patients receiving ianalumab 300 mg monthly showed greater reductions in ESSDAI score compared with placebo (−6.5 vs −5.3; 

 = .0031) and improvements in Patient Global Assessment (−13.5 vs −8.7; 

 = .0049). Both studies also reported consistent benefits across secondary endpoints, including low systemic disease activity, oral dryness, and patient-reported outcomes, with a safety profile comparable to placebo.

REGENCY: Obinutuzumab’s Deep B-Cell Depletion in Kidney Supports Benefit for Lupus Nephritis

Obinutuzumab, a type 2 anti-CD20 antibody, showed profound intrarenal B-cell and plasma cell depletion in lupus nephritis in an exploratory biopsy analysis from the REGENCY trial. 

At Week 76, kidney B-cells fell by a median −98.34% with obinutuzumab versus a +29.8% increase on placebo, and plasma cells dropped by 57.13% compared with a 2.74% rise on placebo. Clinically, 46.4% of patients receiving obinutuzumab plus standard therapy achieved complete renal response versus 33.1% with standard therapy alone.

Low-Dose IL-2 Rebalances Immunity in SLE, With Yuebo Jin, MD

Low-dose interleukin-2 (IL-2) demonstrated clinically meaningful improvements in systemic lupus erythematosus (SLE) in a phase 2b trial. 

At 1.0 million IU, 60% of patients achieved SRI-4 response criteria compared with 23% on placebo, with additional benefits across skin, hematologic, and renal domains. Treatment also increased complement levels, reduced corticosteroid requirements, and showed lower infection rates, highlighting a favorable safety profile while restoring immune balance.

ACR 2025: Osteoarthritis Late-Breakers to Know

Apoptotic Cell Therapy Shows Promising Benefits for Older Adults With Knee OA, with Philip Conaghan, MBBS, PhD

Apoptotic cell therapy, an investigational immunomodulatory cell-based treatment, showed promising efficacy in older adults with moderate-to-severe knee osteoarthritis in the phase 2a ENX-CL-05-001 trial. 

At 3 months, patients ≥60 years achieved a 72% relative improvement in WOMAC pain (–27.82 vs –16.22; 

 = .03) and a 109% improvement in function (–26.45 vs –12.63; 

 = .007) over placebo. Benefits were even stronger in patients ≥65 years, with total WOMAC improving –27.14 vs –6.03 (

 = .0004), and safety findings were mainly transient effusions occurring more often with treatment.

Adipose-Derived MSCs Show Promise as Nonsurgical Option for Knee OA, With Moshiur Rahman Khasru, MBBS, MSc

Two-dose adipose-derived mesenchymal stem cells (AdMSC) therapy showed superior improvements in pain, function, and cartilage structure compared with hyaluronic acid (HA) in patients with knee osteoarthritis (KOA). 

At 6 months, two-dose AdMSC reduced VAS pain by 3.2 points versus 1.3 points with HA and improved KOOS function by 21.24 versus 11.31 points (

 < .001). Structural benefits were also observed, with femoral cartilage thickness increasing by 0.25 mm and MRI-assessed cartilage defects improving by 9.13% in the two-dose AdMSC group, compared with cartilage thinning (−0.06 mm) and defect worsening (0.75%) with HA.

ACR 2025: Fibromyalgia Late-Breakers to Know

Gut Microbiome Modulation by Fiber Enhances RA Methotrexate Response, With Claire Daien, MD, PhD

Fiber supplementation improved clinical response rates and immune balance in rheumatoid arthritis in the randomized SUPERFIBRE trial. 

By Day 30, 53.85% of patients receiving fiber achieved a EULAR response versus 21.74% on placebo (

= .03) after adjustment. Among methotrexate-treated patients, mean ΔDAS28 was −1.00 with fiber compared with 0.34 with placebo (

= .011), and treatment was well tolerated with >90% compliance.

TENS With Physical Therapy Meaningfully Reduced Fibromyalgia Movement-Evoked Pain

Transcutaneous electrical nerve stimulation (TENS), a non-invasive neuromodulation approach, improved movement-evoked pain and related symptoms when added to routine physical therapy in the pragmatic FM-TIPS trial. 

At Day 60, MEP was lower with PT+TENS (mean −1.1; 95% CI, −1.58 to −0.7) and 41% achieved ≥30% MEP reduction versus 13% with PT alone (

 = .0001). Resting pain improvements were also greater (30% vs 21%; 

= .004), with benefits across fatigue measures and no serious treatment-related adverse events.

AgAIN: Secukinumab Outperforms Ustekinumab in Post-TNFi Psoriatic Arthritis, With Frank Behrens, MD

Secukinumab, an IL-17A–inhibiting biologic, showed superior efficacy to ustekinumab in TNFi-experienced PsA in the head-to-head AgAIN trial. 

At Week 28, 57.1% of secukinumab-treated patients achieved a HAQ-DI response versus 27.0% with ustekinumab (OR 3.647; 95% CI, 1.601–8.311; 

 = .002). Secukinumab also delivered faster and broader improvements across joints, skin, pain, and enthesitis, while the ustekinumab group had higher discontinuation rates due to loss of efficacy.

Achieving “State Zero”: New Data on Bimekizumab, Other IL-17is, Transform PsA Treatment Goals, with Philip Mease, MD

New data on IL-17–targeting therapies, including bimekizumab, sonelokimab, and izokibep, highlight evolving treatment strategies in PsA.

Patients achieving “state zero” with bimekizumab, defined as complete resolution of swollen joints, enthesitis, and radiographic progression, experienced substantial improvements in pain, function, and quality of life.

ROC-SpA: IL-17i Not Superior to Cycling TNFi Post-Failure in AxSpA

Switching to an IL-17 inhibitor did not outperform cycling to a second TNF inhibitor in axial spondyloarthritis in the phase 4 ROC-SpA trial. 

At week 24, ASAS40 responses were similar between IL-17i and TNFi groups (15.2% vs 14.5%; 

 not significant), and no secondary endpoints showed statistical differences. Subgroup trends hinted at potential advantages for IL-17i in patients with primary nonresponse, psoriasis, HLA-B27–negative status, or low CRP, but overall disease activity improvements were comparable, with mean ASDAS reaching 2.5 in both groups.

Mepolizumab Demonstrates Benefit for End Organ Dysfunction in EGPA and HES

Mepolizumab, an anti–IL-5 biologic, demonstrated improvements across cardiac, renal, and vascular systems in patients with eosinophilic granulomatosis with polyangiitis (EGPA) and hypereosinophilic syndrome (HES) in a large real-world analysis.

 In patients with EGPA, mepolizumab significantly improved acute kidney failure (

 = .026) and chronic kidney disease stage 3 (

 = .009) compared with standard-of-care therapy. Among patients with HES, mitral regurgitation (

 = .009) were also reduced, highlighting its effectiveness in reducing end-organ dysfunction across multiple systems.

Rosnilimab: A New Era of Upstream RA Disease Control, With Jonathan Graf, MD

Rosnilimab, a novel monoclonal antibody targeting pathogenic T cells, demonstrated rapid and meaningful clinical improvements in patients with moderate to severe rheumatoid arthritis (RA) in a phase 2b trial. 

At week 12, all rosnilimab dosing arms achieved significantly greater reductions in DAS28-CRP compared with placebo (–2.06 to –2.12 vs –1.69; 

 < .01). Improvements were also seen across ACR20/50/70 responses and CDAI low disease activity, with benefits sustained through week 28 and maintained off therapy through week 38.

ACR Convergence 2025 in Chicago presented significant advancements in rheumatology, with late-breaking trials highlighting promising treatments for various conditions. Telitacicept and ianalumab showed efficacy in Sjögren's disease, while obinutuzumab and low-dose IL-2 offered benefits for lupus nephritis and SLE, respectively. Apoptotic cell therapy and adipose-derived MSCs demonstrated potential for knee osteoarthritis. In fibromyalgia, fiber supplementation and TENS improved outcomes. Secukinumab outperformed ustekinumab in PsA, and rosnilimab showed promise in RA. Mepolizumab improved end-organ dysfunction in EGPA and HES, while IL-17 inhibitors were comparable to TNFi in axial spondyloarthritis.

A compilation of 13 trials presented as late-breaking data at ACR Convergence 2025.

Rheumatology

Rheumatology condition center page is a comprehensive resource for clinical news and insights on rheumatologic disease. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for arthritis, gout, nr-AxSpA, and more.

Lupus

Psoriatic Arthritis

On the HCPLive psoriatic arthritis page, resources on the topics of medical news and expert insight into PsA can be found. Content includes articles, interviews, videos, podcasts, and breaking news on arthritis, and more.

Osteoarthritis

On the HCPLive osteoarthritis page, resources on the topics of medical news and expert insight into OA can be found. Content includes articles, interviews, videos, podcasts, and breaking news on degenerative joint disease, and more.

ACR Convergence 2025 Recap: Late-Breaking Trials to Know

Alex Hillenbrand is an assistant editor for 

. She earned her BA in English from Lafayette College in 2023 and went on to write for pop culture, entertainment, and sports media outlets before joining MJH Life Sciences in 2025. In her free time, Alex enjoys cooking, going on walks, and meeting new people. Contact her at mjhlifesciences.com.

The United States Food and Drug Administration (US FDA)

 has approved new safety warnings for Sarepta’s delandistrogene moxeparvovec-rokl (ELEVIDYS) for Duchenne muscular dystrophy (DMD), restricting the indication to ambulatory patients.

According to a November 14, 2025, FDA communication, the therapy received substantial labeling revisions, including a Boxed Warning describing the risk of serious liver injury, acute liver failure, or fatal outcomes.

“The FDA is requiring the manufacturer to conduct a postmarketing observational study to further assess the risk of serious liver injury,” said the FDA in a statement. “The study will enroll approximately 200 patients with DMD and follow them for at least 12 months after administration of Elevidys, with periodic liver function assessments.”

Delandistrogene moxeparvovec-rokl was the first approved gene therapy to treat DMD, a rare, progressive genetic disorder, for use in patients ≥4 years of age. The single-dose, adeno-associated virus-based gene therapy is designed to address the lack of dystrophin protein driven by mutations in the dystrophin gene (DMD).

“We want to thank the FDA for their thorough and collaborative review,” said Louise Rodino-Klapac, PhD, the president of research & development and technical operations at Sarepta Therapeutics. “Completion of the safety labeling change for ELEVIDYS will ensure that families and healthcare professionals have clear information, supported by a Medication Guide, to help understand these updates and guide treatment decisions.”

The therapy received initial FDA approval in 

 based on results from the global, randomized, double-blind, placebo-controlled phase 3 EMBARK trial, which met a surrogate endpoint of increased microdystrophin in individuals 4-5 years of age with DMD and no significant pre-existing antibody titers or contraindications.

In June 2025, following 2 reports of fatal acute liver failure in non-ambulatory pediatric males with DMB who had received delandistrogene moxeparvovec-rokl, the FDA issued a CBER Safety Communication and halted distribution of the drug.

Sarepta released a statement in response, emphasizing patient safety and well-being as their top priority in July 2025 and noting delandistrogene moxeparvovec-rokl as the only approved gene therapy available to patients impacted by DMD. At the time, the drug had been indicated for use in ambulatory and non-ambulatory patients with a confirmed mutation in the DMD gene.

Subsequent investigation into the 2 reported fatal cases showed markedly elevated liver enzymes and hospitalization 2 months post-treatment. A non-fatal case of acute liver injury showed mesenteric vein thrombosis, bowel ischemia/ necrosis, and portal hypertension.

The November 2025 FDA update includes additional safety-specific information and monitoring recommendations for clinicians and patients. The updated guidance recommends weekly liver monitoring for ≥3 months post-treatment and for patients to remain near an appropriate medical facility for ≥ 2 months after infusion.

The new labeling emphasizes that corticosteroid therapy may suppress immune function, increasing susceptibility to infections and serious complications including death and advises weekly testing for troponin-I to detect cardiac injury for 1 month following treatment. Patients are advised to contact a clinician if they exhibit yellowing of the skin or eyes, miss or vomit corticosteroid doses, or notice a change in mental status.

The therapy is not recommended for use in individuals with preexisting liver impairment, recent vaccinations, or recent/active infections, or patients with deletions involving 

FDA Approves New Safety Warning and Revised Indication that Limits Use for Elevidys Following Reports of Fatal Liver Injury. 

. Published online November 14, 2025. Accessed November 14, 2025. 

https://www.fda.gov/news-events/press-announcements/fda-approves-new-safety-warning-and-revised-indication-limits-use-elevidys-following-reports-fatal?utm_medium=email&utm_source=govdelivery

Sarepta Therapeutics Provides Clarifying Statement on ELEVIDYS | Sarepta Therapeutics, Inc. Sarepta Therapeutics, Inc. Published 2025. Accessed November 14, 2025. 

https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-clarifying-statement-elevidys

Sarepta Announces FDA’s Approval of Updated ELEVIDYS Prescribing Information | Sarepta Therapeutics, Inc. Sarepta Therapeutics, Inc. Published 2025. Accessed November 14, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-announces-fdas-approval-updated-elevidys-prescribing

Johnson V. FDA Approves Sarepta’s Landmark DMD Gene Therapy Elevidys. Cgtlive.com. Published June 22, 2023. Accessed November 14, 2025. 

https://www.cgtlive.com/view/fda-approves-sarepta-s-landmark-dmd-gene-therapy-elevidys

The US FDA has updated safety warnings for Sarepta's delandistrogene moxeparvovec-rokl (ELEVIDYS), a gene therapy for Duchenne muscular dystrophy (DMD), limiting its use to ambulatory patients due to risks of severe liver injury. A postmarketing study will assess these risks further. The therapy, initially approved in 2023, faced distribution halts after fatal liver failure cases. New guidelines recommend rigorous liver monitoring and caution against use in patients with certain conditions. The updated labeling also highlights potential immune suppression and cardiac risks.

The FDA adds a boxed warning to delandistrogene moxeparvovec-rokl (ELEVIDYS) restricting indications to ambulatory patients with DMD.


FDA News

Neurology

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FDA Issues New Boxed Warning, Limiting ELEVIDYS to Ambulatory DMD

Vogelmeier discusses major changes to the annual COPD report, including a broader threshold for escalating treatment after exacerbation.

Pulmonology

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COPD

COPD condition center page, resources on the topics of medical news and expert insight into chronic obstructive pulmonary disease can be found. Content includes articles, interviews, videos, podcasts, and breaking news on COPD research, treatment, and drug development.

Breaking Down the 2026 GOLD Report, with Claus Vogelmeier, MD

Chelsie Derman is an associate editor for 

who covers psychiatry, sleep medicine, geriatrics, migraine, and allergy. She joined MJH Life Sciences in September 2023 after graduating from The College of New Jersey with a bachelor’s degree in journalism and professional writing. In her free time, she enjoys creative writing and reading.

Here, we spotlighted 4 late-breaker studies featured in our ACAAI 2025 coverage, along with 2 phase 3 trials. 

Allergy

Allergy condition center page is a comprehensive resource for clinical news and insights on allergic and inflammatory diseases. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for food and seasonal allergies, as well as allergic asthma and related conditions.

Top Trial Recaps from ACAAI 2025

Ron Vender, MD, is a dermatologist in Hamilton, Ontario, and is the founder and director of Dermatrials Research Incorporated and Venderm Consulting Inc. He is also an associate clinical professor at McMaster University in the Department of Medicine, Division of Dermatology.

After his sessions on TYK2 Inhibitors for psoriasis and on palmoplantar pustulosis, Vender discussed several key insights in this interview.

Plaque Psoriasis

The HCPLive plaque psoriasis page is a resource for medical news and expert insights on psoriasis. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for psoriatic disease, and more.

Discussing Zasocitinib and Other TYK2 Inhibitors for Psoriasis, With Ronald Vender, MD

ALA Spokesperson and professor at Baylor College of Medicine.

COPD is not a complex disease, Polverino explains. It's only complex to treat because of limited understanding.

The Practical Hurdles in COPD Detection, with Francesca Polverino, MD, PhD

In this Q&A, Soong discusses phase 2 results on povorcitinib, a JAK1 inhibitor that provides rapid relief from chronic spontaneous urticaria symptoms.

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