Is givinostat’s entry into Japan a turning point for DMD care and for JCR Pharmaceuticals’ rare disease strategy?
Find out how Italfarmaco and JCR are bringing the DMD drug givinostat to Japan and what it means for rare disease innovation in Asia.
Pharma & Biotech
Pharma & Biotech
NeoImmuneTech takes over Endari’s U.S. rights from Emmaus: What this signals for the future of sickle cell treatment
NeoImmuneTech takes over U.S. rights for Endari from Emmaus Life Sciences. Find out what this deal reveals about the future of sickle cell treatment.
Pharma & Biotech
PTC Therapeutics receives Japanese approval for Sephience in full-spectrum PKU
Japan approves Sephience for PKU across all ages. Find out what this means for PTC Therapeutics' global rare disease expansion strategy.
Medical Devices & Diagnostics
Wasatch BioLabs, Agilent to scale native-read targeted sequencing for precision multi-omics
Wasatch BioLabs has announced a co-marketing agreement with Agilent Technologies to expand access to its Direct Targeted Methylation Sequencing (dTMS) platform. The partnership integrates Agilent’s SureSelect and Avida enrichment chemistries with Wasatch BioLabs’ Oxford Nanopore-based native-read workflow, aiming to streamline high-throughput targeted sequencing for research use only (RUO) and translational settings. The announcement marks a
Pharma & Biotech
Waskyra approved for Wiskott-Aldrich syndrome: What it means for stem cell alternatives
Fondazione Telethon has received regulatory approval from the United States Food and Drug Administration for Waskyra (etuvetidigene autotemcel), an ex vivo gene therapy designed to treat Wiskott-Aldrich syndrome, a rare X-linked primary immunodeficiency. This approval makes Waskyra the first gene therapy for this condition cleared for use in the United States and solidifies Fondazione Telethon’s