Immunine (factor IX concentrate (human)) / Takeda - LARVOL DELTA

Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B. (PubMed, N Engl J Med) - P1 | "A single administration of scAAV2/8-LP1-hFIXco gene therapy resulted in durable factor IX expression, sustained clinical benefit, and no late-onset safety concerns over a period of 13 years. These data support the long-term efficacy and safety of AAV gene therapy for severe hemophilia B. (Funded by the U.K. Medical Research Council and others; ClinicalTrials.gov number, NCT00979238; EudraCT number, 2005-005711-17.)."

Journal • Cardiovascular • Fibrosis • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Hepatology • Immunology • Liver Failure • Oncology • Rare Diseases • Thrombosis

A Post-Authorization Safety Surveillance Study to Report Clinical Experience with Purified Factor IX Concentrate in Pediatric Patients with Hemophilia B. (PubMed, J Blood Med) - "Purified factor IX (FIX) concentrate (IMMUNINE®, Takeda Manufacturing Austria AG, Vienna, Austria) is indicated for the treatment and prophylaxis of bleeding episodes in patients with congenital hemophilia B. Data on the use of purified FIX concentrate in patients ≤6 years old with congenital hemophilia B are limited...Hemostatic efficacy was rated as either "excellent" or "good" in all patients with an available rating. No treatment-related AEs were reported, and purified FIX concentrate was shown to be effective in treating and preventing bleeding episodes in pediatric patients ≤6 years old with hemophilia B."

Journal • Hematological Disorders • Hemophilia • Pediatrics • Rare Diseases

[VIRTUAL] HEALTH EDUCATION: PREPARATION OF EDUCATIONAL VIDEOS FOR THE RECONSTITUTION OF COAGULATION FACTORS (HEMO 2021) - "Production: The recording of the videos was performed by a qualified professional, in a recording studio, with a presenter actress, a resident nurse in hematology, with a duration of four days. On the first day, photography of the products was taken; on the second day there was a recording of the actress' performance; on the third day, the audios were recorded and added to the video images; on the fourth day, the necessary changes were made for completion. Four types of coagulation factors were filmed (recombinant factor VIII, plasma factor VIII, immunine factor IX and grifols factor IX), ending a total time of 5 minutes and 16 seconds. engage in the construction and use of new technologies in their area of ​​expertise, both in teaching and in practice. The development of studies and strategies in health education aims to facilitate user learning, encouraging the modification of health habits and seeking achieve the benefits of its..."

Genetic Disorders • Hematological Disorders • Hemophilia • Pain • Rare Diseases • Rheumatology

[VIRTUAL] Real World Experience of GlycoPEGylated Recombinant Factor IX concentrate in Patients with Moderate and Severe Haemophilia B in the UK (ISTH 2020) - "Full abstracts will be available June 29, 2020 at 9:00 AM U.S. Eastern Time"

Clinical • Real-World Evidence • Gene Therapies • Genetic Disorders • Hematological Disorders • Hemophilia • Rare Diseases