Latest Conference Articles

Bernaudin discussed the Drepagreffe-1 and 2 studies and improvements seen over 10 years of follow-up.

Real-world clinical data from the Adelphi PNH II Disease Specific Programme were reported at ASH 2024.

Danicopan add-on therapy demonstrated more favorable safety in the ALPHA trial than pegcetacoplan did in the PEGASUS trial in a new analysis.

Appiah-Kubi also noted that despite elevated HbF levels, very young children with SCA already had anemia and reticulocytosis.

Pipe discussed findings from the open-label extension of the ATLAS studies at ASH 2024.

At ASH 2024, Annette von Drygalski, MD, PharmD, described the paradigm shift that has taken place in hemophilia over the past decade.

Piatek shared the latest data update from the phase 3 ALPHA study at the ASH 2024.

Frangoul discussed highlights from the latest data update from the CLIMB SCD-121 trial of Casgevy.

Data from the PINES trial at ASH 2024 suggest eltrombopag could outperform current standard of care for platelet response in newly diagnosed pediatric ITP.

A propensity score-matched study from ASH 2024 suggests GLP-1 RA use reduced VTE risk in type 2 diabetes.

While the therapy was generally well-tolerated, the PIVOT trial failed its primary endpoint of dose-limiting toxicities.

The therapy uses a novel pyruvate kinase activator mechanism and warrants further investigation in an ongoing phase 3 trial.

Notably, participants without platelet responses also experienced improvements in fatigue, which may be due to the monoclonal antibody’s anti-inflammatory action.

Schattenberg reviews results from a posthoc analysis of the ENLIVEN study suggesting pegozafermin’s impact on preventing progression to cirrhosis.

Pharmacokinetics and immunogenicity were also similar between reference tocilizumab and CT-P47.

Schattenberg explains pegozafermin’s mechanism of action and describes the importance of preventing progression to cirrhotic MASH.

Our Cardiology Month in Review for November 2024 spotlights the approval of acoramidis and top content from AHA 2024.

Test your knowledge of the 2024 AASLD Practice Guidelines on blood- and imaging-based noninvasive liver disease assessment with this clinical quiz!

Test your knowledge of the AASLD NAFLD Practice Guidance and its clinical application in light of the new MASLD nomenclature.

David G. Armstrong, DPM, PhD, and Michael S. Conte, MD discuss Phase 2 results on anatomically directed lower extremity gene therapy for ulcer healing.

Fromme describes the importance of risk stratification in AATD and explains the potential utility of noninvasive fibrosis measures for predicting outcomes.

Strnad reviews findings from a proteomic analysis about AATD-associated liver disease and how they may aid future disease monitoring and drug development.

Hirschfield describes the need for therapies that address itch in PBC and explains what linerixibat, an IBAT inhibitor, may offer for pruritus.

Kavish Patidar, DO, explains how socioeconomic status and healthcare delivery impact outcomes in patients with acute kidney injury and cirrhosis.

Kugelmas reviews findings from a secondary analysis of the phase 3 ELATIVE trial suggesting elafibranor is associated with stable kidney function.

Birgit Vogel, MD discusses racial inequities and geographic variations in peripheral artery disease care in the United States.

Lawitz explains issues with traditional FXR agonists for MASH treatment and how FXR314 differs, potentially offering a more safe and efficacious option.

Milind Desai, MD describes favorable real-world safety outcomes from mavacamten use over 22 months of the Risk Evaluation and Mitigation Strategy (REMS) program.