Medical director, pediatric hematology/oncology, Sarah Cannon Pediatric Transplant and Cellular Therapy Program, TriStar Centennial, and investigator, Sarah Cannon Research Institute

What Clinicians Should Discuss Before Exa-cel Gene Therapy

Following the US Food and Drug Administration (FDA)’s expanded approval of exagamglogene autotemcel (exa-cel; Casgevy) for children ≥2 years of age with sickle cell disease (SCD) and recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β-thalassemia (TDT), the next challenge will be helping patients and families understand what undergoing gene therapy actually entails.

“I think we need to be more thoughtful about explaining the gene therapy journey, what I call it, because it's truly a journey,” Haydar Frangoul, MD, MS, director of the pediatric stem cell transplant program at Sarah Cannon Research Institute and TriStar Centennial Children’s Hospital and first author of multiple landmark exa-cel studies told 

Unlike an allogeneic bone marrow transplant, where a donor provides stem cells for a recipient, exa-cel uses a patient’s own hematopoietic stem cells that are collected, genetically modified, and returned after treatment.

“The therapy is long, so unlike a bone marrow transplant, when you have a donor and you have a recipient and you can bring them in, and you can do a transplant within few weeks,” explained Frangoul. “Here, you have to collect the cells, you have to manufacture them.”

The manufacturing process adds several steps before infusion, requiring patients and families to understand that treatment involves a longer timeline than traditional transplantation.

In the phase 3 pediatric studies supporting the expanded indication, children underwent collection of their own stem cells followed by ex vivo CRISPR/Cas9 editing and myeloablative conditioning with busulfan before receiving exa-cel.

How to Counsel Patients on Myeloablative Conditioning

Before receiving exa-cel, patients undergo myeloablative conditioning to prepare the bone marrow for the edited cells. For many individuals with SCD or TDT, this may be their first experience with intensive chemotherapy.

“Unlike our patients that we treat, for example, in the setting of cancer therapy, where they get a bone marrow transplant, they have received chemo, they have lost their hair, they have developed complications related to chemotherapy. Here, in this therapy, people with sickle cell disease and thalassemia have never received chemo.”

Frangoul emphasized that counseling should include both the immediate effects of chemotherapy and the temporary immune suppression associated with conditioning.

“The discussion should include the short-term side effect of chemotherapy, which includes the loss of your hair, the development of mouth sores, the immune suppression as far as decreasing the immune system.”

While exa-cel avoids risks associated with donor transplantation, including graft-versus-host disease and graft rejection, conditioning-related toxicities remain an important consideration. In the pediatric phase 3 study, all participants experienced ≥ 1 grade 3 or 4 adverse event. Two children with TDT developed severe veno-occlusive liver disease considered related to busulfan conditioning, including 1 death.

Beyond short-term toxicities, Frangoul highlighted fertility as one of the most important long-term considerations before beginning therapy.

“The big one is infertility. So there is a higher risk of infertility with using busulfan chemotherapy, and individuals, parents, and children and adults considering this therapy need to consider whether they would want to pursue fertility preservation before this therapy.”

Because exa-cel is now being considered for younger patients, including children and adolescents, fertility preservation discussions may become an increasingly important part of treatment planning.

For clinicians, these conversations provide an opportunity to help families weigh the potential benefits of gene therapy alongside the risks and practical considerations involved in undergoing treatment.

Editor’s Note: Frangoul reports relevant disclosures with Vertex Pharmaceuticals, Editas Medicine, Rocket Pharmaceuticals, and others. 

FDA approves first gene therapy fsor young children with sickle cell disease.

Frangoul H, Locatelli F, Sharma A, et al. Exagamglogene autotemcel for severe sickle cell disease. 

 2024;390(18):1649-1662. doi:10.1056/NEJMoa2309676.

Frangoul H, et al. Exagamglogene autotemcel in children with severe sickle cell disease and transfusion-dependent β-thalassemia. 

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Exa-cel requires careful counseling on treatment timelines, chemotherapy conditioning, fertility risks, and the gene therapy journey.

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FDA News

Counseling Patients On Exa-cel Gene Therapy Journey, With Haydar Frangoul, MD, MS

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.

 receiving long-term prophylaxis (LTP) who treated breakthrough attacks with oral sebetralstat achieved beginning of symptom relief in a median 1.3 hours, according to an interim analysis of the KONFIDENT-S open-label extension study, with effectiveness consistent regardless of which prophylactic agent patients were taking.¹

The prespecified analysis, published in the 

Journal of Allergy and Clinical Immunology: Global

, evaluated 35 participants with HAE due to C1-inhibitor deficiency (HAE-C1INH) who were receiving LTP with berotralstat (n = 16), lanadelumab (n = 13), or C1 inhibitor (C1INH) replacement (n = 6) while enrolled in the 2-year KONFIDENT-S extension of the phase 3 KONFIDENT trial.¹ Despite background prophylaxis, these participants experienced a mean ± standard deviation of 1.7 ± 1.5 breakthrough attacks per month and treated 382 of 504 total breakthrough attacks with on-demand sebetralstat 600 mg, the oral plasma kallikrein inhibitor approved by the US Food and Drug Administration (FDA) in July 2025 as the first oral on-demand HAE therapy, the second of 3 HAE treatments approved in successive months from June to August.¹⁻²

“In the span of just 3 months, there were 3 new treatment modalities approved in HAE, which is mind-blowing, really, but it's really a testament to all the work that's gone into [the field] in the last two decades. If we think back 16 years ago, that’s when the launch of modern-era medicine came out for hereditary angioedema and and to think of how far we’ve come in the treatment landscape just makes my other colleagues and other specialties envious of the advances that we have made,” Raffi Tachdjian, MD, MPH, from UCLA Health, previously told 

Investigators, led by Tamar Kinaciyan, MD, of the Medical University of Vienna, found that median time from attack recognition to sebetralstat administration was just 6 minutes (interquartile range [IQR], 1-40), a finding the authors attributed to the study's protocol-driven instruction to treat as early as possible.¹ Nearly 30% of attacks were treated while still mild. Median time to reduction in attack severity was 4.2 hours (IQR, 1.3 to >12), and median time to complete attack resolution was 14.8 hours (IQR, 4.6 to >24).¹ A post hoc analysis of time to end of attack progression, added after the primary effectiveness end points were defined, showed a median of 20.4 minutes, with 91% of sebetralstat-treated attacks reaching end of progression within 4 hours.¹

Effectiveness was broadly similar across attack severities, anatomic locations, and participant age groups, and did not differ meaningfully between participants on plasma kallikrein-inhibiting LTP (berotralstat or lanadelumab) and those on C1INH replacement.¹ Hazard ratios comparing participants receiving LTP with those receiving on-demand treatment alone were 1.38 (95% CI, 0.93-2.04) for time to symptom relief, 1.53 (95% CI, 0.98-2.40) for time to reduction in severity, and 1.75 (95% CI, 1.03-2.99) for time to complete resolution; investigators characterized effectiveness as "generally consistent" between the 2 groups, though these were unadjusted comparisons within a descriptive, open-label dataset rather than a powered statistical test.¹

A second sebetralstat dose was administered within 12 hours in 22.3% of attacks, and conventional injectable on-demand therapy was used as rescue in only 5.2% of sebetralstat-treated attacks, suggesting a single oral dose was sufficient for most breakthrough episodes.¹ Angioedema Quality of Life (AE-QoL) scores trended toward improvement through month 15, with a least squares mean change from baseline of -10.3 (95% CI, -21.1 to 0.6) — a confidence interval that crosses the null, meaning the improvement did not reach statistical significance at that time point.¹ Treatment satisfaction was high: participants reported being satisfied or better with sebetralstat for 88.8% of 276 rated attacks.¹

Safety was consistent with the drug's established profile. Of the 35 participants receiving LTP, 65.7% experienced a treatment-emergent adverse event (TEAE), and 14.3% experienced a TEAE considered treatment-related, most commonly headache, myalgia, arthralgia, nausea, or vomiting.¹ No serious or severe treatment-related TEAEs occurred. Two participants discontinued sebetralstat because of adverse events — 1 for a TEAE later attributed to viral meningitis and hepatotoxic comedications rather than sebetralstat, and 1 for nausea and vomiting during a mixed abdominal/laryngeal attack, in which gastrointestinal drug effects can be difficult to distinguish from attack symptoms.¹

The authors acknowledged several limitations relevant to interpreting the findings. The open-label design, while reflective of real-world use and permitting a larger attack sample than the randomized KONFIDENT trial, introduces potential reporting bias.¹ The analysis was descriptive and explicitly not powered to detect differences by LTP agent or across subgroups, and the C1INH replacement cohort was particularly small (n = 6), limiting how generalizable those findings are to that population.¹ The study was funded by KalVista Pharmaceuticals, sponsor and manufacturer of sebetralstat; several authors reported consulting fees, honoraria, or research support from the company, and 3 authors are former employees and shareholders while 3 others are current employees and shareholders.¹ Medical writing assistance was also funded by KalVista.¹

Sebetralstat's role alongside targeted LTP has drawn increasing clinical attention as HAE guidelines shift toward earlier, patient-controlled on-demand treatment regardless of background prophylaxis.³ The KONFIDENT-S data build on the smaller LTP subgroup from the original KONFIDENT trial, which included 24 participants receiving targeted LTP who treated 58 attacks, and now offer a considerably larger real-world-like dataset to inform how clinicians counsel patients who continue to break through on prophylaxis.¹⁻²

Kinaciyan T, Aygören-Pürsün E, Martinez-Saguer I, et al. Sebetralstat for breakthrough attacks in patients with hereditary angioedema receiving long-term prophylaxis in KONFIDENT-S. J Allergy Clin Immunol Global. 2026;5:100750. https://doi.org/10.1016/j.jacig.2026.100750

Riedl MA, Farkas H, Aygören-Pürsün E, et al. Oral sebetralstat for on-demand treatment of hereditary angioedema attacks. N Engl J Med. 2024;391:32-43. https://www.nejm.org/doi/10.1056/NEJMoa2314192

FDA Approves Sebetralstat for Hereditary Angioedema. AJMC. https://www.ajmc.com/view/fda-approves-sebetralstat-for-hereditary-angioedema

Articles

Interim KONFIDENT-S open-label extension data in HAE-C1INH patients on long-term prophylaxis (berotralstat, lanadelumab, or C1INH replacement) showed oral sebetralstat 600 mg provided rapid on-demand control of breakthrough attacks. Median time to symptom relief was 1.3 hours, with dosing occurring quickly after recognition (median 6 minutes). Median time to severity reduction and complete resolution were 4.2 and 14.8 hours, respectively, with broadly similar effectiveness across prophylaxis classes and subgroups. Safety reflected the established tolerability profile.

Interim KONFIDENT-S data show breakthrough HAE attacks began to be relieved at a median of 1.2 hours.

Allergy

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Sebetralstat Shows Long Term Fast Relief of Breakthrough HAE Attacks

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.

has expanded the approved use of Ryaltris (olopatadine hydrochloride and mometasone furoate) nasal spray, 665 mcg/25 mcg per spray, to include the treatment of 

symptoms in children aged 6 to 11 years, Glenmark Pharmaceuticals announced July 21, 2026.

With the approval, Ryaltris is now indicated for adults and pediatric patients aged ≥ 6 years in the United States, broadening access to a younger population than the drug's original 2022 approval for patients aged ≥ 12 years.

Ryaltris combines olopatadine hydrochloride, an antihistamine, with mometasone furoate, a corticosteroid, in a single nasal spray formulation.

“This approval marks an important step in expanding access to Ryaltris in the United States,” said Marc Kikuchi, president & business head in North America, in a statement.

Phase 3 Trial Data Supporting Ryaltris Pediatric Approval

The expanded indication was supported by a phase 3, randomized, double-blind, placebo-controlled trial of 446 children aged 6 to 11 years with seasonal allergic rhinitis.¹ Investigators found that Ryaltris. delivered as 1 spray in each nostril twice daily, improved nasal symptoms compared with placebo and was well tolerated over the 14-day treatment period.

Seasonal allergies remain common among school-age children in the US. According to the Centers for Disease Control and Prevention, 25% of children aged 6 to 11 years were more likely to have a diagnosed seasonal allergy than children aged 0 to 5 years in 2024.

Ryaltris Safety Profile and Warnings for Pediatric Use

Ryaltris is contraindicated in patients with known hypersensitivity to its ingredients, including mometasone furoate. Warnings include risk of epistaxis, nasal ulcerations, septal perforation, and impaired wound healing.

The label also notes risks of hypersensitivity reactions, immunosuppression with worsening of existing infections, hypercorticism and adrenal suppression with misuse, and potential reduction in growth velocity in pediatric patients. The most common adverse reactions, occurring in at least 1% of patients, are dysgeusia, epistaxis, and nasal discomfort.

Glenmark Receives U.S. FDA Approval to Expand the Use of RYALTRIS® Nasal Spray for Children Aged 6 to Less Than 12 Years. Glenmark. Published July 21, 2026. Accessed July 21, 2026. 

https://innovacia.in/glenmark-receives-u-s-fda-approval-to-expand-ryaltris-use-for-children-aged-6-to-less-than-12-years/

Ryaltris FDA Approval History. Drugs.com. Published on January 20, 2022. Accessed on July 21, 2026. 

https://www.drugs.com/history/ryaltris.html

Prenner BM, Amar NJ, Hampel FC Jr, Caracta CF, Wu W. Efficacy and safety of GSP301 nasal spray in children aged 6 to 11 years with seasonal allergic rhinitis. 

National Center for Health Statistics. Diagnosed Allergic Conditions in Children Ages 0–17: United States, 2024. NCHS Data Brief No. 546. January 2026. 

https://www.cdc.gov/nchs/products/databriefs/db546.htm

The FDA expanded Ryaltris (olopatadine hydrochloride/mometasone furoate) nasal spray to treat seasonal allergic rhinitis in children aged 6–11 years, extending its US indication to patients aged ≥6 years. The fixed-dose antihistamine–intranasal corticosteroid combination was previously approved for ages ≥12 years. A phase 3 randomized, double-blind, placebo-controlled trial in 446 children showed twice-daily dosing (one spray per nostril) improved nasal symptoms versus placebo over 14 days with acceptable tolerability. Labeling emphasizes local nasal injury risks, infection-related precautions, and potential pediatric growth effects.

FDA expands RYALTRIS® approval to children aged 6 to 11 with seasonal allergic rhinitis, based on phase 3 trial data.

Seasonal Allergy

FDA Approves Ryaltris for Children Aged 6 to 11 With Seasonal Allergic Rhinitis

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.

Topline results from a phase 2 trial of tebapivat in patients aged ≥16 years with

showed improvements in hemoglobin and markers of hemolysis but did not demonstrate sufficient differentiation to support continued development of the pyruvate kinase (PK) activator, prompting Agios to discontinue the program.

"These phase 2 data further reinforce PK activation as a clinically validated mechanism in sickle cell disease, with tebapivat demonstrating hematologic activity consistent with this class of medicine," said Sarah Gheuens, MD, PhD, chief medical officer and head of research and development at Agios, in a statement. "However, the results did not establish the level of differentiation we believe is necessary to support continued development."

 12-week, randomized, double-blind, placebo-controlled phase 2 trial

 ≥1.0 g/dL increase in hemoglobin during Weeks 10-12

The phase 2 trial was designed to characterize the dose-response relationship of tebapivat in SCD and evaluate whether the therapy demonstrated a differentiated profile relative to other PK activators.

The 12-week, double-blind, placebo-controlled study randomized 59 patients in a 2:2:2:1 ratio to receive once-daily tebapivat at doses of 2.5 mg, 5.0 mg, or 7.5 mg or placebo QD.

The primary endpoint was hemoglobin response, defined as a ≥1.0 g/dL increase in average hemoglobin concentration during Weeks 10 through 12 compared with baseline.

According to Agios, the primary endpoint was achieved by 43.8% (n = 7/16), 47.1% (n = 8/17), and 29.4% (n = 5/17) of patients receiving 2.5 mg, 5.0 mg, and 7.5 mg tebapivat, respectively, compared with 33.3% (n = 3/9) receiving placebo.

The company also reported improvements in hemoglobin levels and markers of hemolysis across tebapivat dose groups, findings it said were consistent with the established mechanism of PK activation. Safety and tolerability were consistent with prior SCD studies.

Although tebapivat demonstrated hematologic activity, Agios stated that the phase 2 findings did not establish the level of differentiation it considered necessary to support continued development in SCD. The company did not cite safety or tolerability concerns as a reason for discontinuing the program.

PK activators increase pyruvate kinase activity in red blood cells to improve cellular energy metabolism, with the goal of reducing hemolysis and improving anemia in patients with SCD. Agios noted that the results further support PK activation as a clinically validated therapeutic mechanism despite its decision not to advance tebapivat.

Agios said it will continue to prioritize mitapivat, its lead oral PK activator, which is currently under 

Earlier this month, the Agency accepted the company's supplemental New Drug Application (sNDA) for mitapivat and assigned a Prescription Drug User Fee Act (PDUFA) target action date of November 1, 2026.

FDA Grants Priority Review to Agios’ Mitapivat sNDA for Sickle Cell Disease

"We remain focused on mitapivat, our foundational PK activator, which is under FDA Priority Review in sickle cell disease with an expected US approval later this year," Gheuens said. "We look forward to bringing this first-in-class medicine to the sickle cell community and building on the extensive clinical experience generated to date as we work to address the significant unmet need in this debilitating disease."

Agios Provides Update on Phase 2 Trial of Tebapivat in Sickle Cell Disease – Agios Pharmaceuticals, Inc. (2026). Agios Pharmaceuticals, Inc. 

https://investor.agios.com/news-releases/news-release-details/agios-provides-update-phase-2-trial-tebapivat-sickle-cell

US Food and Drug Administration. Pyrukynd (mitapivat) prescribing information. Accessed July 21, 2026. 

https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/216196s000lbl.pdf

Topline phase 2 data for tebapivat in patients ≥16 years with sickle cell disease showed modest hemoglobin improvements and favorable shifts in hemolysis markers, consistent with pyruvate kinase activation. In a 12-week, randomized, double-blind, placebo-controlled design, hemoglobin response (≥1.0 g/dL increase at Weeks 10–12) was numerically higher in the 2.5 mg and 5.0 mg arms versus placebo, but not clearly dose-responsive and less compelling at 7.5 mg. With no new safety signal, development was halted due to insufficient differentiation.

Agios ends tebapivat development in sickle cell disease after phase 2 results show no differentiated benefit.

Agios Ends Tebapivat Development in Sickle Cell Disease After Phase 2

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.

Shady Abohashem, MD, MPH | Image Credit: LinkedIn

The majority of US adults are significantly above LDL-C goals, according to an analysis including the 2026 American College of Cardiology/American Heart Association 

The updated 2026 guidelines included the reintroduction of the Predicting Risk of Cardiovascular Disease EVENTs (PREVENT) equations, replacing the existing pooled cohort equations (PCEs), as well as establishing risk-based LDL-C treatment goals. These updated goals prompted a reevaluation of the proportion of patients still above new LDL-C goals to estimate the prevalence of dyslipidemia and its ensuing conditions in the US.

The present study, conducted by Shady Abohashem, MD, MPH, the head of cardiac PET/CT imaging trials and an instructor at Harvard Medical School, and colleagues, seeks to examine the degree to which US adults are exceeding their LDL-C goals – and how many are receiving lipid-lowering therapy to reverse this.

Abohashem and colleagues gathered data from the National Health and Nutrition Examination Survey (NHANES) 2021-2023 cycle, prioritizing adults aged 30-79 years. Response rates were 25.7% - the team adjusted survey weights for nonresponse, and no evidence was found regarding systematic nonresponse bias.

atherosclerotic cardiovascular disease (ASCVD)

 risk among patients without self-reported ASCVD or heart failure via the PREVENT base equations. The collected risk data were then categorized as low (<3%), borderline or intermediate (3-<10%), or high (≥10%). Established ASCVD was defined via self-reported coronary heart disease, myocardial infarction, angina, or stroke, while very high risk was assigned through a combination of major ASCVD events and high-risk conditions as determined by NHANES data.

Abohashem and colleagues derived LDL-C from venous blood collected during the morning fasting subsample of the NHANES mobile examination visit. Reference goals were assigned by the patient’s risk category, with <160 mg/dL for low-risk adults, <100 mg/dL for borderline adults, <70 mg/dL for high-risk adults, <70 mg/dL for adults with ASCVD not meeting very high risk criteria, and <55 mg/dL for adults with ASCVD meeting very high risk criteria. Secondary analyses determined lipid-lowering therapy use among patients above LDL-C goals via self-reported use of prescription medication for high cholesterol.

A total of 2313 patients were included in the analytic sample, representing roughly 177.7 million adults (87.8% in primary prevention, 12.2% in secondary). Among the primary prevention cohort, roughly 32.2% had LDL-C above guideline-recommended goals (95% CI, 29.6-34.9%). Prevalence increased among higher risk categories.

Among low-risk adults, 9.9% were above goal. 63.6% of borderline/intermediate risk adults were above goal, and 82.7% were above goal among high-risk adults. The proportion above goal was notably higher among older, male adults. Of adults with established ASCVD, 79.6% were above goal (95% CI, 73.5-84.8%). Among adults above goal, roughly 76.1% (95% CI, 71.1-80.6%) in primary prevention and 37.6% (95% CI, 29.9-45.8%) in secondary prevention were not receiving lipid-lowering therapy.

Abohashem and colleagues attributed the observed treatment gaps to a variety of factors, including clinical inertia, limited patient awareness, and barriers to adherence or access. Additionally, while primary prevention patients’ next step was statin initiation, secondary prevention typically included patients already on lipid-lowering therapy and required intensification with higher-intensity statins or nonstatin therapies.

The team also reported potential limitations to the study, including reduced NHANES response rates and participant self-report of ASCVD status and use of lipid-lowering therapy. Additionally, the AHA/ACC guidelines do not explicitly state an LDL-C goal for low-risk adults.

“These estimates reflect contemporary lipid levels prior to release of the guideline, and future studies are needed to evaluate how adoption of the updated guideline may influence lipid management and cardiovascular outcomes in practice,” Abohashem and colleagues wrote.

Abohashem S, Martin S, Hassan I, et al. Prevalence of LDL-C Above 2026 Dyslipidemia Guideline Goals Among US Adults. 

. July 20, 2026. Accessed July 20, 2026. 

Blumenthal RS﻿, Morris PB﻿, Gaudino M﻿, et al. 2026 ACC/AHA/AACVPR/ABC/ACPM/ADA/AGS/APhA/ASPC/NLA/PCNA guideline on the management of dyslipidemia: a report of the American College of Cardiology/American Heart Association Joint Committee on Clinical Practice Guidelines. 

. March 13, 2026. Accessed July 20, 2026. 

https://doi.org/10.1161/CIR.0000000000001423

Updated 2026 ACC/AHA dyslipidemia guidance reintroduced PREVENT risk equations and set risk-based LDL-C goals, prompting reassessment of goal attainment in US adults. Using NHANES 2021–2023 fasting labs in adults aged 30–79 years, LDL-C thresholds were assigned by primary- and secondary-prevention risk strata. An estimated 32.2% in primary prevention exceeded targets, with sharp gradients across risk categories. In established ASCVD, 79.6% were above goal, alongside substantial undertreatment, particularly in primary prevention.

According to a recent study, nearly 32.2% of US adults are well above their LDL-C targets to avoid atherosclerotic cardiovascular disease.

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Dyslipidemia

Atherosclerotic Cardiovascular Disease

New Dyslipidemia Guidelines Reveal Significant Gap in LDL-C and LLT Treatment Goals

After broadening eligibility criteria across several disease states and a wider age range, the new guidelines have made an estimated 87.5 million adults eligible for statins.

Millions of New Patients Eligible for Statins Under 2026 AHA/ACC Guidelines

Stay updated with the latest healthcare breakthroughs, including FDA approvals, phase 3 data, and open-label extension results.

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The HCPFive: Top News for Healthcare Providers from the Week of 07/12

John Sonnenberg, PhD, clinical psychologist, founder of Uptown Research Institute, and faculty at Northwestern University Feinberg School of Medicine.

John Sonnenberg, PhD, outlines EMERGE trial criteria, dosing sessions, and endpoints for DT120 (lysergide) ODT 100 µg in major depressive disorder.

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Sonnenberg Breaks Down EMERGE Phase 3 Trial Design for DT120 in MDD

Haydar Frangoul, MD, discusses how the expanded indication may allow earlier conversations about curative therapy and intervention before irreversible complications develop.

FDA Expands Exa-Cel (Casgevy) Approval, Bringing Gene Therapy to Younger Children

Ron Blankstein, MD, is the associate director of the cardiovascular imaging program, director of cardiac computed tomography, and co-director of the cardiovascular imaging training program at Brigham and Women’s Hospital, as well as a professor of medicine at Harvard Medical School.

Ron Blankstein, MD, discusses a secondary analysis of HeartFlow’s DECIDE registry, indicating undetected cardiovascular risk in patients previously labeled safe.

Total Plaque Volume Indicates Patients With 0 CAC May Still be at Risk

Kimberly Blumenthal, MD, discusses who qualifies for de-labeling and how risk stratification guides testing.

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Most Penicillin Allergy Labels Are Wrong, Blumenthal Says

Richard Lafayette, MD, is a professor of medicine and director of the Glomerular Disease Center at Stanford University Medical Center.

ORIGIN 3 investigator brings atacicept approval to BAFF/APRIL targeting to IgAN 

IgA Nephropathy

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

Atacicept Approval Brings BAFF/APRIL Targeting to IgAN With Richard Lafayette, MD

Abigail Brooks is a strategic content lead overseeing 

She joined MJH Life Sciences in August 2023 shortly after graduating from Monmouth University where she earned her BA in Communication with a concentration in Public Relations/Journalism and later an MA in Interactive Digital Media. She enjoys traveling, running, and reading books. She can be reached at abrooks@mjhlifesciences.com.

Newly published phase 2b RELIEVE UCCD data show strong 14 week results for duvakitug in ulcerative colitis and Crohn disease.

Gastroenterology

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

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TL1A Antibody Duvakitug Boosts Clinical Remission in UC, Response in CD

Ashish Rana, MD, is the internal medicine program director and associate chair of academic medicine at Crozer Health, as well as a member of Sermo’s advisory board.

Ashish Rana, MD, discusses Sermo’s recent clinician survey, which highlighted patients’ use of unapproved and compounded GLP-1 medications.

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This page also hosts content previously published by EndocrinologyNetwork. 


Survey Shows Peptide Misinformation, Misuse Rising in US

Kimberly Blumenthal, MD, MSc, explains how surgical dyes, antibiotic-coated implants, and more can trigger perioperative anaphylaxis without appearing in the chart.

Latest News

Counseling Patients On Exa-cel Gene Therapy Journey, With Haydar Frangoul, MD, MS

Sebetralstat Shows Long Term Fast Relief of Breakthrough HAE Attacks

FDA Approves Ryaltris for Children Aged 6 to 11 With Seasonal Allergic Rhinitis

Agios Ends Tebapivat Development in Sickle Cell Disease After Phase 2

New Dyslipidemia Guidelines Reveal Significant Gap in LDL-C and LLT Treatment Goals