Victoria Johnson

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.

Chaturvedi discussed findings from the NeST study at ASH 2024, including an increased risk of stroke in those with silent cerebral infarction progression.

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.

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.

The biosimilars month in review highlights new equivalency data for denosumab and tocilizumab biosimilars.

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

Phase 1/2 data prompted the RMAT designation for HLHS shortly after Mesoblast announced positive phase 3 data for HFrEF.

Masri discussed the new phase 3 trial of Intellia’s NTLA-2001 or nex-z.

HCPLive takes a look at one of the newer fields investigating gene therapy, which has been enabled by trials and approvals in hematology, ophthalmology, and other spaces.

This nephrology month in review spotlights HCPLive’s coverage of ASN Kidney Week, the debut of a new podcast, and new research on IgA nephropathy.

If approved, HLX14 would follow the first 2 denosumab biosimilar approvals in March 2024.

MASEF score seemed to be sensitive and effective in detecting at-risk MASH in 2 abstracts presented at the ACG 2024 Meeting.

Direct-acting antiviral agents have enabled more availability for organ transplant.

The oral therapy may address limitations including difficulty in application and adverse events with topical therapies.

New PRO data from the INTEGUMENT-1 and –2 studies were presented at the ACAAI 2024 Meeting.

The biosimilars month in review highlights new approvals, new safety findings, and new cost savings with biosimilars.

Mice models had improved disease phenotype and high transduction of PS-002 gene therapy.

Yee discussed research she is conducting into gFSGS and nephrotic syndrome, presented at ASN Kidney Week.

Abdellatif discussed the high prevalence of gout in patients with chronic kidney disease and the importance of screening and early treatment.

Zand discussed the unmet need for refractory primary FSGS and the importance of investigations specifically in this population.

Complete, 24-month data from the IGNAZ study were presented at the ASN Kidney Week 2024 Meeting.

The FDA originally issued a CRL requesting additional CMC data in April 2024.

Refardt discussed findings from the largest hyponatremia trial ever conducted, including 2174 participants across 9 European centers.

The dapagliflozin group had an average eGFR rate of -2.24 mL/min/1.73 m2 compared to -3.67 mL/min/1.73 m2 in the control group after about 1.5 years of follow-up.