Chelsie Derman is an associate editor for 

who covers allergy and  psychiatry. 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.

Credit: Children’s Hospital of Philadelphia

In a prospective cohort study of 100 children and young adults with 

 treated at Children's Hospital of Philadelphia, 94% of initiation challenges were tolerated after a median of 12 weeks of omalizumab (Xolair) pretreatment, allowing most patients to begin oral immunotherapy (OIT) directly at maintenance dosing.¹

Standard OIT protocols require multistep dose escalations and frequent in-office up-dosing visits, creating a substantial treatment burden for patients with multi-food allergy. Investigators evaluated whether a short course of omalizumab pretreatment could allow most patients to skip incremental up-dosing and begin OIT directly at maintenance.

Omalizumab-Facilitated OIT Study Design and Initiation Outcomes

Colleen Shannon, MD, MPH, from the Children’s Hospital of Philadelphia, and colleagues conducted a prospective cohort study of 100 children and young adults who initiated omalizumab-facilitated OIT between July 2024 and October 2025.¹ Patients received ≥ 8 weeks of omalizumab pretreatment before undergoing a 2-dose initiation challenge for each target food. If tolerated, daily OIT dosing began at maintenance for most foods, with abbreviated up-dosing reserved for the remaining foods.

Median omalizumab pretreatment duration was 12 weeks (interquartile range [IQR], 9.5-15.8 weeks). Patients tolerated 181 of 193 initiation challenges (94%), meeting the primary outcome of tolerance without dose-limiting symptoms. In total, 97 patients (97%) initiated daily dosing for ≥ 1 food, and 81 patients (84%) started at target maintenance doses across all foods in their regimen.

These findings indicate the omalizumab-facilitated protocol allowed most patients to bypass the incremental dose-escalation phase required by conventional OIT.

Safety and Follow-Up Outcomes With Omalizumab-Facilitated OIT


Omalizumab-facilitated OIT pairs a short course of omalizumab pretreatment with oral immunotherapy, raising the threshold for allergic reactions before food dosing begins.

How effective was direct initiation at maintenance dosing in this cohort?


Patients tolerated 94% (181 of 193) of initiation challenges after a median of 12 weeks of omalizumab pretreatment, and 84% started at target maintenance doses for all foods.


The most common adverse events were abdominal pain (23%) and oral pruritus (13%), with 2 patients discontinuing therapy because of adverse events.

During median follow-up of 27.6 weeks (IQR, 18.1-41.1 weeks), 39 patients (40%) reported ≥ 1 adverse event.¹ The most common events were abdominal pain, reported by 23% of patients, and oral pruritus, reported by 13%. In total, 2 patients discontinued OIT because of adverse events.

Among patients who met criteria for omalizumab discontinuation, median treatment duration was 26.3 weeks (IQR, 20.7-36.4 weeks). Investigators characterized the overall safety profile as acceptable, with treatment discontinuation uncommon across the cohort.

Abdominal pain and oral pruritus are commonly reported with OIT dose escalation, and their frequency in this cohort was consistent with expectations for a multi-food regimen. Omalizumab has an established safety profile across its approved indications, including IgE-mediated food allergy, moderate-to-severe asthma, chronic urticaria, and chronic rhinosinusitis with nasal polyps.² The low discontinuation rate observed here suggests the direct-to-maintenance approach did not introduce new tolerability concerns beyond those seen with standard OIT.

“This approach may reduce treatment burden while maintaining an acceptable safety profile,” investigators concluded.

A separate randomized trial found omalizumab produced significantly greater treatment success than omalizumab-facilitated multiallergen oral immunotherapy (MOIT) in patients with multifood allergy, per stage 2 results from the OUTMATCH trial.³ Among 117 participants with peanut allergy and ≥ 2 additional food allergies, omalizumab was superior to MOIT for the primary endpoint of cumulative tolerated dose in the intention-to-treat population (36% vs 19%; odds ratio, 2.6; 95% CI, 1.1-6.3; 

 =.03). This difference narrowed in the per-protocol population, reflecting a substantially higher discontinuation rate in the MOIT arm (49%) compared with omalizumab (12%).

MOIT also carried a heavier safety burden, with more serious adverse events (31% vs 0%), epinephrine-treated reactions (37% vs 7%), and events meeting anaphylaxis criteria (27% vs 2%) compared with omalizumab.³

"Both treatments can be effective, and both will provide protection from small exposures and even the ability to start eating the food directly," Robert A. Wood, MD, professor of pediatrics, Johns Hopkins University School of Medicine, told 

.³ "The biggest difference we found was the side effects from oral immunotherapy were such a far larger group of patients really couldn't tolerate the therapy.”

Shannon CM, Kennedy KL, Lee J, Hanna E, Spergel J, Cianferoni A. Omalizumab-facilitated oral immunotherapy with direct initiation at maintenance dosing for most foods. 

Smith T. FDA Approves Omalizumab as First Treatment of 1 or More Food Allergies for Children, Adults. HCPLive. Published February 16, 2024. Accessed August 7, 2026. 

https://www.hcplive.com/view/fda-approves-omalozumab-first-treatment-of-1-or-more-food-allergies-for-children-adults

Wood R. Omalizumab Outperforms Oral Immunotherapy for Multifood Allergy. HCPLive. Published July 29, 2026. Accessed August 7, 2026. 

https://www.hcplive.com/view/omalizumab-outperforms-oral-immunotherapy-multifood-allergy

Articles

A prospective cohort of 100 pediatric and young adult patients with multifood allergy used ≥8 weeks of omalizumab pretreatment to enable oral immunotherapy initiation largely at maintenance dosing. After a median 12-week pretreatment, 94% of 193 two-dose initiation challenges were tolerated; 97% began daily dosing for at least one food, and 84% reached maintenance across all foods. Over ~28 weeks’ follow-up, adverse events were typical for OIT and rarely led to discontinuation. Separate randomized data favored omalizumab over multiallergen OIT due to tolerability.

A prospective cohort study found 94% of patients tolerated direct initiation at maintenance OIT dosing after brief omalizumab pretreatment.

News

news page, resources on the topics of disease- and specialty-specific medical news and expert insight can be found. Content includes articles, interviews, videos, podcasts, and breaking news on health care research, treatment, and drug development.

Clinical

clinical page is a resource for content on every specialty, disease, and topic available on our medical news site.

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.

Food Allergy

Omalizumab-Facilitated OIT Enables Maintenance Dosing in Food Allergy

Tim Smith joined the MJH Life Sciences team in 2022 and is currently an associate editor for 

. He is also the executive producer of two podcasts: 

. 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.

• Socrodeucitinib 12 mg once daily achieved PASI 75 in 72.1% of patients at week 12 versus 7.5% with placebo (

 <.001).
• Responses were dose-dependent across PASI 50, PASI 90, PASI 100 and sPGA 0/1 outcomes, all statistically significant versus placebo at 12 mg.
• Adverse events were more common with socrodeucitinib, most frequently upper respiratory tract infection, but serious adverse events and discontinuations were infrequent.
• No deaths, major adverse cardiovascular events, malignancies or clinically meaningful laboratory abnormalities were reported.
• The 12 mg once-daily dose has advanced into an ongoing phase 3 trial (NCT06672393).

Socrodeucitinib, an oral tyrosine kinase 2 (TYK2) inhibitor, achieved significantly higher rates of psoriasis area and severity index (PASI) 75 response at Week 12 compared with placebo in a phase 2 randomized trial of individuals with 

Journal of the European Academy of Dermatology and Venereology

TYK2 inhibitors have emerged as a differentiated oral option in the systemic psoriasis landscape, positioned as more selective than JAK1/2/3 inhibitors, which carry boxed warnings for cardiovascular events, malignancy and infection. Deucravacitinib and TAK-279 previously demonstrated the clinical potential of pseudokinase-directed TYK2 inhibition, and socrodeucitinib (HS-10374) adds phase 2 data supporting a 12 mg once-daily dose ahead of an ongoing phase 3 program.²

Socrodeucitinib Trial Design and PASI 75 Results

The phase 2, randomized, double-blind, placebo-controlled trial enrolled 125 patients across 38 centers in China between September 2023 and April 2024. Patients were stratified by prior biologic use and randomized 1:1:1 to receive oral socrodeucitinib at 6 mg, 12 mg or placebo once-per-day for 12 weeks.

Eligible adults were 18 to 70 years old with plaque psoriasis for 6 months or longer, a static Physician's Global Assessment (sPGA) score of 3 or higher, a PASI score of 12 or higher and body surface area involvement of 10% or greater. Baseline characteristics were balanced across arms, with a mean PASI score of 20.0 and 26.4% of patients previously treated with a biologic agent.

At Week 12, PASI 75 was achieved by 72.1% of patients on the 12 mg dose and 28.6% on the 6 mg dose, compared with 7.5% on placebo (12 mg, 

 = .013). The proportion difference versus placebo was 65.2 percentage points at the 12 mg dose (95% CI, 50.07-80.34) and 21.2 percentage points at the 6 mg dose (95% CI, 5.47-36.96). Responses emerged as early as Week 4 and were sustained through Week 12 at both doses.

Socrodeucitinib Secondary Endpoints and Safety Profile

Secondary endpoints tracked the primary result at the 12 mg dose. PASI 90 was achieved by 46.5% of patients versus 0% on placebo (P <.001), and PASI 100 by 11.6% versus 0% (

PASI 50 was reached by 81.4% versus 20.0% on placebo (

 <.001), and an sPGA score of 0 or 1 by 65.1% versus 10.0% on placebo (

 <.001). Dermatology Life Quality Index scores improved by a least-squares mean of 6.9 points at the 12 mg dose versus 0.2 points on placebo (

 <.001), with separation from placebo apparent by Week 4.

Adverse events occurred in 88.4% of patients on the 12 mg dose, 76.2% on the 6 mg dose and 70.0% on placebo, with most graded mild to moderate. Upper respiratory tract infection was the most frequent adverse event and showed a dose-dependent pattern, occurring in 23.3% of patients on the 12 mg dose versus 16.7% on the 6 mg dose and 7.5% on placebo.

Serious adverse events were infrequent, reported in one patient on placebo and one on the 6 mg dose, with none on the 12 mg dose. No deaths, major adverse cardiovascular events, thromboses or malignancies were reported, and laboratory parameters showed no clinically meaningful abnormal trends versus placebo.

Based on the efficacy and exposure-response findings, the 12 mg once-daily dose has been selected for an ongoing phase 3 trial (NCT06672393) in patients with moderate to severe plaque psoriasis.

Han L, Geng S, Ding Y, et al. A randomized phase 2 trial of socrodeucitinib in moderate-to-severe plaque psoriasis. J Eur Acad Dermatol Venereol. 2026;00:1-9. 

Chinese Clinical Trial Registry. A randomized, double-blind, placebo-controlled, phase 2 study of socrodeucitinib in patients with moderate to severe plaque psoriasis. CTR20232241. Accessed August 7, 2026.

Socrodeucitinib, an oral TYK2 inhibitor, demonstrated dose-dependent clinical activity in moderate-to-severe plaque psoriasis over 12 weeks in a multicenter China-based phase 2 trial. PASI 75 responses were substantially higher with 12 mg once daily versus placebo, with separation by Week 4 and sustained benefit through Week 12. Higher-stringency endpoints (PASI 90/100), sPGA 0/1, and DLQI improvements similarly favored 12 mg. Adverse events were mostly mild-to-moderate, with dose-related upper respiratory tract infections and no observed MACE, thrombosis, malignancy, or deaths.

Oral socrodeucitinib 12 mg significantly improved PASI 75 and sPGA responses versus placebo at Week 12 in a 125-patient phase 2 trial.

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.

Socrodeucitinib Meets Primary Endpoint in Phase 2 Psoriasis Study

Marc Bonaca, MD, MPH, is the executive director at CPC Clinical Research, director of vascular research at the University of Colorado Anschutz, and a cardiologist with Brigham and Women’s Hospital and the VA Boston Healthcare System.

The phase 3 VESALIUS-CV trial has shown that evolocumab protects against cardiovascular disease in at-risk patients with 

 prior to screening, hinting at a new treatment pathway for cardiovascular disease.

VESALIUS-CV: Evolocumab Prevents Cardiovascular Disease in High-Risk Diabetes

These data were discussed at the American Society for Preventive Cardiology (ASPC) 2026 Congress on Cardiovascular Disease Prevention in Scottsdale, Arizona, by Marc Bonaca, MD, MPH, executive director at CPC Clinical Research, director of vascular research at the University of Colorado Anschutz, and a cardiologist with Brigham and Women’s Hospital and the VA Boston Healthcare System.

“VESALIUS moved backwards in terms of risk,” Bonaca told 

 in an exclusive interview. “It included patients who were either at high risk without known 

, or those who had it but who had not yet had an event. VESALIUS really asked the question of whether PCSK9 inhibition benefits patients who are at high risk or have the disease but haven’t yet experienced an event.”

VESALIUS-CV was a double-blind, randomized, placebo-controlled, multicenter phase 3 study examining evolocumab’s impact on MACEs in patients at high cardiovascular risk without prior MI or stroke. The study was conducted across 852 locations worldwide and ultimately enrolled over 6000 patients. To be eligible for inclusion, patients needed to be ≥50 years (men) or ≥55 years (women) and <80 years and have LDL-C ≥90 mg/dL, non-HDL-C ≥120 mg/dL, or apolipoprotein B ≥80 mg/dL. Additionally, patients were required to meet ≥1 of the following at screening:

Significant coronary artery disease (CAD)

Significant atherosclerotic cerebrovascular disease

Patients were excluded from the trial if they had experienced an MI or stroke prior to randomization, had undergone coronary artery bypass grafting (CABG) <3 months before surgery, or eGFR <15 mL/min/1.73m

, among other criteria. The primary outcomes were the number of patients experiencing coronary heart disease (CHD), MI, or ischemic stroke, whichever occurred first, as well as the number of patients experiencing CHD death, MI, ischemic stroke, or any ischemia-driven arterial revascularization, whichever occurred first.

The team ultimately enrolled 6002 patients with high-risk diabetes. Of these, 67% were on a high-intensity statin, and 24% were on an SGLT2 inhibitor or a GLP-1 receptor agonist at baseline. Median LDL-C at 48 weeks was 47 mg/dL and 109 mg/dL in the evolocumab and placebo arms, respectively (

Following a median follow-up of 4.6 years, evolocumab was found to have decreased the relative rates of 3-point MACE and 4-point MACE by 29% (HR, 0.71; 95% CI, 0.59-0.86; 

 = .0004) and 21% (HR, 0.79; 95% CI, 0.69-0.91; 

 = .0013), respectively. Additionally, these findings were consistent irrespective of the presence or absence of atherosclerosis, baseline LDL-C, statin treatment intensity, and SGLT2 inhibitor or GLP-1 RA use. Investigators noted that the proportion of patients with all-cause mortality was 8.8% in the evolocumab arm versus 11% in the placebo arm (HR, 0.79; 95% CI, 0.67-0.93).

Ultimately, investigators concluded that evolocumab effectively reduced the rate of cardiovascular events in this patient cohort, regardless of the background use of other protective agents or the presence or absence of qualifying atherosclerosis.

“The field is shifting as we become much more sophisticated about understanding atherosclerosis,” Bonaca said. “If you think about it, for cancer screenings, we don’t wait for people to become symptomatic to treat them, and we don’t treat them based on guessing whether or not they have disease. We image – we conduct colonoscopies, mammograms, biopsies, and we treat accordingly. And for the number one killer in the world, cardiovascular disease, we now have tools that allow a much more sophisticated approach.”

Editors’ Note: Bonaca reports disclosures with Abbott, Amgen, BMS, Merck, NovoNordisk, Sanofi, and others.

Leiter LA, Giugliano RP, Marston NA, et al. Evolocumab in patients with high-risk diabetes: Results from the vesalius-CV trial. Diabetes Care. 2026;49(8):1366-1373. 

Amgen. Effect of Evolocumab in Patients at High Cardiovascular Risk Without Prior Myocardial Infarction or Stroke (VESALIUS-CV). ClinicalTrials.gov Identifier: NCT03872401. Updated June 25, 2026. Accessed 

https://clinicaltrials.gov/study/NCT03872401

Videos

The phase 3 trial of evolocumab showed cardioprotective effects in patients without prior MI or stroke, regardless of qualifying atherosclerosis or background therapy.

Cardiology

Cardiology condition center page is a comprehensive resource for clinical news and insights on cardiovascular and cardiometabolic diseases. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for heart disease and cardiovascular events, as well as associated diabetes, renal failure, and more...

Stroke

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

Structural Heart Disease

Endocrinology

The HCPLive endocrinology page is a comprehensive resource for clinical news and insights on endocrine system conditions. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for diabetes, hormonal disease, and more.

This page also hosts content previously published by EndocrinologyNetwork. 


Type 2 Diabetes

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.

A new consensus framework for treating hepatocellular carcinoma (HCC) outperformed the current staging system in a pilot study. BEACON-HCC matched independent expert treatment decisions 96.6% of the time, compared with 72.4% for the Barcelona Clinic Liver Cancer (BCLC) 2025 system. The framework was published by the American Association for the Study of Liver Diseases (AASLD) in 

Framework Developed to Reflect Expanding HCC Treatment Landscape

BEACON-HCC was developed by 20 North American experts spanning hepatology, medical oncology, surgery, radiology, and radiation oncology. The consensus was created using a modified Delphi process and is intended to update treatment allocation approaches for HCC, which investigators note have not kept pace with the growing number of available therapies.

HCC remains the third leading cause of cancer-related death worldwide and the leading cause of cancer-related mortality among patients with cirrhosis. Five-year survival remains below 25%, a statistic the framework's developers cite as justification for a more contemporary approach to treatment planning.

“HCC treatment has evolved dramatically over the past decade, yet many existing treatment frameworks have not fully incorporated these advances,” said Amit G. Singal, MD, MS, FAASLD, of University of Texas Southwestern, in a statement. “BEACON-HCC reflects how multidisciplinary experts evaluate patients today by integrating the latest evidence with real-world clinical expertise to support more personalized treatment decisions.”

What Sets BEACON-HCC Apart From Existing Staging Systems

Rather than relying solely on liver function and disease stage, BEACON-HCC incorporates tumor biology, overall disease burden, and extent of vascular invasion into its recommendations. The framework also accounts for a broader set of contemporary treatment modalities, including external beam radiation therapy (EBRT), transarterial radioembolization (TARE), and combined systemic-locoregional strategies.

“The treatment landscape for liver cancer is changing rapidly,” said Mark Yarchoan, MD, of the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Medicine, in a statement. “This framework provides clinicians with a contemporary roadmap that better reflects current evidence, multidisciplinary expertise, and the expanding therapeutic options available to patients.”

The concordance figures cited above come from a pilot study of 29 real-world patient cases, in which independent multidisciplinary experts not involved in developing BEACON-HCC reviewed cases and made treatment recommendations. 

Those decisions were then compared against both the BEACON-HCC framework and BCLC 2025 recommendations to calculate concordance. Given the small sample size, the authors describe the pilot as an initial validation step rather than definitive evidence.

Clinical Implications and Next Steps for Validation

The authors describe BEACON-HCC as a complement to, rather than a replacement for, multidisciplinary clinical decision-making, and note that it will continue to be refined as new evidence emerges. Further validation is planned through the HCC-Live Consortium using prospective clinical data.

The publication is being positioned as one of the more comprehensive multidisciplinary efforts to redefine HCC treatment allocation in North America to date.

Singal AG, Yarchoan M, et al. BEACON-HCC: Best Evidence and North American Consensus on Hepatocellular Carcinoma. 

. Published online August 5, 2026. doi:10.1097/HEP.0000000000001834

American Association for the Study of Liver Diseases. HEPATOLOGY publishes landmark North American framework to advance treatment decisions for liver cancer. Press release. August 7, 2026. Accessed August 7, 2026. 

https://www.aasld.org/hepatology-publishes-landmark-north-american-framework-advance-treatment-decisions-liver-cancer

BEACON-HCC, a North American consensus treatment-allocation framework for hepatocellular carcinoma, showed markedly higher concordance with independent expert recommendations than BCLC 2025 in a 29-case pilot (96.6% vs 72.4%). Developed via a modified Delphi process by a 20-member multidisciplinary panel, it aims to modernize decision pathways amid rapidly expanding systemic and locoregional options and persistently poor outcomes in cirrhosis-associated HCC. The framework integrates tumor biology, disease burden, and vascular invasion, and anticipates iterative refinement with prospective validation.

A new BEACON-HCC framework for liver cancer treatment matched expert decisions 96.6% of the time in a pilot study, outperforming BCLC 2025.

New Liver Cancer BEACON-HCC Framework Shows 96.6% Concordance in Pilot

Stacey Rifkin-Zenenberg, DO, is a Pediatric Hematologist Oncologist in Hackensack, NJ. She has 30 years of experience and is board certified in Hospice Care and Palliative Medicine.

Gene therapy, which uses a patient's own genetically modified cells as a curative treatment, avoiding the need for a matched donor, has produced strong early results for patients with 

, freeing many from the vaso-occlusive crises that previously defined their disease course. Even so, 2 areas of outcome remain under active study: organ function over time, and psychosocial adjustment following treatment.

Stacey Rifkin-Zenenberg, DO, MS, FAAP, FAAHPM, Section Chief of Pediatric Pain and Palliative Care and a pediatric hematologist/oncologist in Hackensack, NJ, discussed both the current landscape and the long-term questions researchers are still working to answer.

With 2 Curative Pathways, Sickle Cell Intervention Can Start in Infancy

After the Cure: What We Still Don't Know About Sickle Cell Gene Therapy

"What we're now seeing is that most of the patients that have received gene therapy are actually transformed," Rifkin-Zenenberg said. "Their sickle cell is transformed. They don't have crises."

At the same time, she pointed to 2 areas she is watching closely as more patients move further out from treatment:

"I'd like to see how they do as they go through time with their clinical organ function, and how they are also doing from a psychosocial point of view."

Rifkin-Zenenberg did not specify which organ systems concern her most, but her point tracks with what the broader literature has found so far. 

Sickle cell disease can cause progressive organ damage well before a curative treatment ever happens, so a patient's post-treatment organ trajectory reflects both that pre-existing burden and whatever longer-term effects gene therapy itself may carry. 

Trials to date have shown durable disease modification, but the toxicity data available so far is largely short-term, meaning long-term organ outcomes are still being collected rather than fully known.

Psychosocial Outcomes After Curative Treatment

A cure doesn't necessarily mean an easy transition. In a mixed-methods study of adult sickle cell disease patients after allogeneic stem cell transplantation, most reported improvements in social health, but emotional struggles and feelings of being overwhelmed were also common, and many wanted more psychological counseling.¹

Whether gene therapy recipients face a similar adjustment is unclear — this evidence comes from transplant patients, and comparable research on gene therapy patients doesn't yet exist.

Tracking Long-Term Outcomes: STELLAR and CureSCi

Two efforts are specifically structured to follow these outcomes over time:

 (Sickle Cell Transplantation Evaluation of Long-term and Late Effects Registry) compares long-term outcomes after transplant against both unaffected siblings and non-transplanted patients with sickle cell disease.

 has developed standardized data elements for gene therapy trials, including patient-reported outcome instruments covering pain, sleep, fatigue, anxiety, depression, and cognition, alongside clinical endpoints such as organ function.

Sickle Cell Disease: Clinical Implications for Curative Treatment

Rifkin-Zenenberg noted that discussion of curative options should begin early, regardless of the current state of long-term outcome data.

For patients and families, that leaves 2 distinct pieces of information: sustained elimination of crises has been observed in most treated patients so far, and organ function and psychosocial outcomes remain under active follow-up rather than fully characterized.

Editor’s Note: Rifkin reports relevant disclosures with Bluebird Bio and Vertex Pharmaceuticals Incorporated.

Sharma A. How I treat sickle cell disease with gene therapy. 

. 2024;144(26):2693-2705. doi:10.1182/blood.2024024519

Tardif M, Saby M, Forté S, Pincez T. The journey of gene therapy in sickle cell disease: how molecular advances meet clinical care. 

. 2026;15(10):939. doi:10.3390/cells15100939

Krishnamurti L, Arnold SD, Haight A, et al. Sickle Cell Transplantation Evaluation of Long-term and Late Effects Registry (STELLAR) to compare long-term outcomes after hematopoietic cell transplantation to those in siblings without sickle cell disease and in nontransplanted individuals with sickle cell disease: design and feasibility study. 

Lanzkron S, Coleman-Cowger VH, Thompson AA, Carroll CP, Clemons T, DeBaun M, Kanter J, Malik P, Manwani D, Pierciey FJ, Walters MC, Alai S. Cure Sickle Cell Initiative recommendations on common data elements for sickle cell disease gene therapy trials. 

. 2026;10(11):3960-3965. doi:10.1182/bloodadvances.2025019400

Stacey Rifkin-Zenenberg, DO, on early gene therapy outcomes and the long-term questions still being tracked in sickle cell disease.

Strategic Alliance Partnership

After Sickle Cell Gene Therapy, What Comes Next?

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.

Examining individual pregnancy outcomes rather than defining women by the presence or absence of complications more reliably predicts later CVD death.

Adverse Pregnancy Outcomes as a Predictor of Cardiovascular Disease Mortality

Roxana Daneshjou, MD, PhD, is a board-certified dermatologist and physician-scientist who serves at Stanford University as an assistant professor in both the Department of Biomedical Data Science and the Department of Dermatology.

A Stanford dermatologist explains how AI systems can reinforce racial bias in health care and underperform on skin cancer detection in darker skin.

Dermatology

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

Podcasts

podcasts page features a feed of medical news and interview podcast series, including DocTalk, Lungcast, Derm Discussions, and Heart Trials.

Does Artificial Intelligence Worsen Health Care Disparities?

Elizabeth Spencer, MD, MS, is an assistant professor of pediatrics in the division of pediatric Gastroenterology at the Icahn School of Medicine at Mount Sinai and at the Mount Sinai Kravis Children’s Hospital.

PFAS found in cord blood linked to childhood gut inflammation and IBD risk; Elizabeth Spencer, MD, explains what it means for patients.

Gastroenterology

Gastroenterology condition center page is a comprehensive resource for clinical news and insights on digestive and GI conditions. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for 

, IBS and IBD, Crohn's disease, and more.

Inflammatory Bowel Disease

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

"Forever Chemicals" Are in Cord Blood And Tied to Gut Inflammation in Kids

In RESOLVE-Lung, the anti-GM-CSF antibody namilumab did not reduce rescue events or improve lung function versus placebo in chronic pulmonary sarcoidosis.

Pulmonology

Infectious Disease condition center page is a comprehensive resource for clinical news and insights on respiratory health. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for asthma, COPD, interstitial lung disease, COVID-19, and more.

Namilumab Fails to Meet Primary End Point in Pulmonary Sarcoidosis

A more precise, endotype-driven understanding of bronchiectasis, paired with an expanding treatment pipeline, is reshaping expectations for what long-term disease control can look like.

Video Series

The Future of Bronchiectasis Care: Precision Medicine Ahead

Turning new bronchiectasis guidelines into everyday practice demands more than awareness, requiring hands-on familiarity with unfamiliar therapies and dedicated staff to share the treatment burden.

Bringing Bronchiectasis Guidelines Into Everyday Practice

Additional DPP1 inhibitors in development show early promise, even as persistent gaps in physician education and validated biomarkers continue to limit optimal bronchiectasis care.

Emerging DPP1 Inhibitors and Closing the Education Gap in Bronchiectasis

With multiple therapeutic classes now available, sequencing macrolides, DPP1 inhibitors, and emerging antibody therapies requires weighing safety, cost, and each patient's underlying disease drivers.

Sequencing Therapies for Bronchiectasis: A Practical Approach

Landmark phase 3 data on DPP1 inhibition mark a shift toward targeting the neutrophilic inflammation that drives bronchiectasis progression, rather than only its downstream consequences.

DPP1 Inhibition in Bronchiectasis: Mechanism and Pivotal Trial Data

Pseudomonas colonization narrows the antibiotic options available for bronchiectasis exacerbations, making inhaled antibiotic strategy a pivotal decision point in long-term management.

Latest News

Omalizumab-Facilitated OIT Enables Maintenance Dosing in Food Allergy

Socrodeucitinib Meets Primary Endpoint in Phase 2 Psoriasis Study

VESALIUS-CV: Evolocumab Prevents Cardiovascular Disease in High-Risk Diabetes

New Liver Cancer BEACON-HCC Framework Shows 96.6% Concordance in Pilot

After Sickle Cell Gene Therapy, What Comes Next?