Higher Risk of ILD in Patients With RA, PsA Initiating bDMARDs
There were no differences in risk between patients with RA or PsA using or not using methotrexate.
Machine Learning Effectively Characterizes Rheumatoid Arthritis Subtypes
Investigators noted that a formal independent validation cohort needs to be developed to realize the full potential of the human model.
Rett Syndrome Gene Therapy Granted RMAT Designation
The FDA granted the Regenerative Medicine Advanced Therapy (RMAT) designation after reviewing the phase 1 safety and efficacy data.
Pfizer’s Hemophilia B Gene Therapy Approved as Beqvez in Canada
The FDA and the European Medicines Agency have accepted marketing authorization applications and decisions are pending.
Excision Bio Seeks to Suppress HIV Replication With CRISPR Gene Therapy
Excision BioTherapeutics has so far released positive safety data from the first 3 participants living with HIV-1, with no evidence of vector shedding in sexual organ tissue.
Kyverna’s CAR T-Cell Therapy Expands Autoimmune Investigations Into Myasthenia Gravis
The therapy is already being evaluated in patients with lupus nephritis and diffuse cutaneous systemic sclerosis.
Patients With T1D Achieve Insulin Independence With Islet Cell Therapy
Updated findings from a phase 1/2 trial of stem-cell derived islet cell therapy for type 1 diabetes (T1D) showed improved glycemic control and a reduction in insulin dependence.
Patients, Clinicians Need Hemophilia Gene Therapy Shared Decision-Making Tool
Clinicians and nurses who treat patients with hemophilia with a gene therapy identified important factors and considerations needed in a potential shared decision making tool.