Find disease awareness content and relevant supporting materials

The FDA is investigating reports of patients developing blood cancer after receiving bluebird bio’s gene therapy Skysona (elivaldogene autotemcel) and is evaluating the need for “further regulatory action,” The FDA notification came seven weeks after the New England Journal of Medicine (NEJM) published a report that seven of 67 children who received Skysona during clinical trials had developed blood cancer

Ipsen  and Biomunex Pharmaceuticals announced an exclusive global licensing agreement for BMX 502. BMX 502 is a bispecific antibody that engages and activates a subset of cytotoxic T cells called Mucosal-Associated Invariant T cells (MAIT cells) and targets the GPC3 tumor antigen, to kill cancer cells

NICE (UK): 1.1 Eplontersen is recommended, within its marketing authorisation, as an option for treating hereditary transthyretin-related amyloidosis in adults with stage 1 or stage 2 polyneuropathy

Cassava Sciences, Inc  announced that the topline results from the Phase III ReThink-ALZ study of simufilam in mild-to-moderate AD did not meet each of the pre-specified co-primary, secondary and exploratory biomarker endpoints

The business combination of two AI-powered drug discovery and development companies, Recursion and Exscientia has been completed, with Exscientia becoming a wholly owned subsidiary of Recursion creating a vertically-integrated and technology-enabled drug discovery platform

Shorla Oncology ’ a U.S.-Ireland specialty pharmaceutical company, announced  that the FDA has approved Imkeldi (imatinib) oral solution, the first oral liquid form of imatinib to treat certain forms of leukemia and other cancers

A pooled analysis of the TROPION-Lung05 phase III and the TROPION-Lung01 phase III trials showed datopotamab deruxtecan (Dato-DXd) demonstrated clinically meaningful tumor response in patients with previously treated advanced or metastatic EGFR-mutated non-small cell lung cancer (NSCLC). These data, along with progression-free and overall survival results from the analysis, were presented during a late-breaking proffered paper session (LBA7) at the 2024 ESMO Asia Congress.

Novartis  announced positive topline results from APPULSE-PNH, a Phase IIIB study evaluating the efficacy and safety of twice-daily oral monotherapy Fabhalta (iptacopan) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who were switched from anti-C5 therapies (Hb ≥10g/dL following treatment with eculizumab or ravulizumab

Patients with nephropathic cystinosis often face a range of gastrointestinal (GI) complications that can severely impact their well-being and treatment adherence. This review aims to provide a comprehensive overview of these GI sequelae, offering insights into their prevalence, pathophysiology, and management strategies to enhance patient care and quality of life.

GSK plc  and Medicines for Malaria Venture (MMV) announced  that the World Health Organization (WHO) has awarded prequalification to tafenoquine, the first single-dose medicine for the prevention of relapse of Plasmodium vivax (P. vivax) malaria