Data from Longest and Largest Hemophilia Gene Therapy Study Show Durable and Sustained Bleed Control and Factor VIII Expression Maintained Four Years Post-ROCTAVIAN Infusion. Additional Data Show Meaningful Impact of ROCTAVIAN on Health-Related Quality of Life (HRQoL). SAN RAFAEL, Calif., June 7, 2024 /PRNewswire/ — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that new data supporting the long-term safety and efficacy of ROCTAVIAN® (valoctocogene roxaparvovec-rvox) will be presented at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Bangkok, Thailand, June 22-26, 2024. The Phase 3 GENEr8-1 trial demonstrated that durable bleed control and sustained factor VIII (FVIII) expression were maintained four years after treatment with ROCTAVIAN, with FVIII activity near stable compared with results reported previously and no new safety signals observed. Of the 134 patients who received ROCTAVIAN in the study, the rollover population… 

Data from Longest and Largest Hemophilia Gene Therapy Study Show Durable and Sustained Bleed Control and Factor VIII Expression Maintained Four Years Post-ROCTAVIAN Infusion. Additional Data Show Meaningful Impact of ROCTAVIAN on Health-Related Quality of Life (HRQoL). SAN RAFAEL, Calif., June 7, 2024 /PRNewswire/ — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that new data supporting the long-term safety and efficacy of ROCTAVIAN® (valoctocogene roxaparvovec-rvox) will be presented at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Bangkok, Thailand, June 22-26, 2024. The Phase 3 GENEr8-1 trial demonstrated that durable bleed control and sustained factor VIII (FVIII) expression were maintained four years after treatment with ROCTAVIAN, with FVIII activity near stable compared with results reported previously and no new safety signals observed. Of the 134 patients who received ROCTAVIAN in the study, the rollover population… 

A new study from deCODE genetics uses pedigrees and sequence data from 64,806 Icelanders to shed light on the rate and nature of mutations in mitochondrial DNA (mtDNA) and the peculiar dynamics of its maternal transmission. In a paper published today in Cell, scientists from deCODE genetics, a subsidiary of Amgen, present the largest study to date of germline mtDNA mutations in humans and their transmission across 116,663 mother-child pairs. The study documents the astonishing extent of hypermutability at some positions in mtDNA, including the well-known deleterious A>G mutation at position 3243 which causes the MELAS syndrome. The mutation occurred 15 times in the 2,548 matrilineal pedigrees, but typically disappeared after several generations, due to its severe impact on the health of carriers. Strong aggregate evidence was uncovered for selection against many such short-lived deleterious mtDNA mutations in the pedigrees.… 

„Baxter wollte sich nicht dazu äußern, ob die in zwei Produkten von Welch Allyn festgestellten Schwachstellen auf einen Verstoß oder einen Cyberangriff zurückzuführen sind.“ Das berichtet MedTech Dive. Die Cybersecurity and Infrastructure Security Agency habe letzte Woche zwei Hinweise zu Sicherheitslücken in Baxter-Produkten veröffentlicht, die es nicht autorisierten Benutzern ermöglichen könnten, Maschinen zu kompromittieren oder auf Anmeldeinformationen zuzugreifen. Die Hinweise beschreiben laut MedTech Dive die Cyberrisiken eines Patientenmonitors und eines Konfigurationstools von Baxter Welch Allyn. Im Hinweis zum Konfigurationstool heiße es, Baxter habe „bisher keine Hinweise auf eine Gefährdung persönlicher oder gesundheitlicher Daten“ gefunden, während im Hinweis zum Patientenmonitor steht, dass CISA bislang keine öffentliche Ausnutzung dieser Schwachstelle gemeldet wurde, „die speziell auf diese Schwachstelle abzielt“. Baxter veröffentlichte dem Bericht zufolge am 30. Mai Bulletins zur Cybersicherheit auf seiner Website und wollte sich nicht dazu äußern, wie die Schwachstellen ermittelt…