San Francisco, CA – Children and adults with rare, deadly genetic diseases have fresh hope for curative therapies, thanks to a new collaboration between the Innovative Genomics Institute (IGI) and Danaher Corporation, a global life sciences and diagnostics innovator. The new Danaher-IGI Beacon for CRISPR Cures center will use genome...
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CAMBRIDGE, Mass. – Biogen Inc. (Nasdaq: BIIB) today announced new research supporting the continued development of an investigational higher dose of SPINRAZA® (nusinersen) and additional data reinforcing the strength of SPINRAZA’s clinical profile in improving the lives of individuals with spinal muscular atrophy (SMA) over the long term. These data...
CAMBRIDGE, Mass. & SINGAPORE – Sunbird Bio, a biotechnology company developing proprietary protein- and blood-based technologies to improve diagnosis and treatment of neurological disorders and early-stage cancer, today announced new data demonstrating that their APEX (Amplified Plasmonic EXosomes) amyloid beta (Aβ) test, the company’s investigational blood-based diagnostic in Alzheimer’s disease,...
ALLSCHWIL, Switzerland – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced results from a post-hoc pooled analysis of the Phase 3 GRIPHON and Phase 3b TRITON clinical trials evaluating the impact of early initiation of UPTRAVI® (selexipag) on disease progression, in a large population of PAH patients. Featured...
Basel – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced results from the final analysis of the phase IIIb STASEY study, which confirm the favourable safety profile of Hemlibra® (emicizumab), consistent with the phase III HAVEN clinical programme.[1],[2],[3],[4] In the analysis, no new safety signals were identified with longer-term Hemlibra...
New data from the phase II CITYSCAPE trial show encouraging results with Roche’s novel anti-TIGIT tiragolumab plus Tecentriq After 2.5 years median follow-up, pre-planned exploratory analyses in the PD-L1-high population show clinically meaningful results, with overall survival not yet reached and continued progression-free survival improvement for the combination compared with...
Ingelheim/Germany — New data presented today at the Movement Disorder Society’s 13th International Congress of Parkinson’s Disease and Movement Disorders (MDS) further confirms high therapeutic benefit of Mirapexin®/Sifrol® (pramipexole) once-daily, extended release formulation (also referred to as prolonged release formulation). The trial, performed in patients with advanced Parkinson’s disease (PD),...
SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced data from the first study on the impact of caring for a child with the ultra-rare genetic disorder, aromatic L-Amino acid decarboxylase deficiency (AADC-d). Results show that caregivers spend almost every waking moment caring for a child with AADC-d,...
SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced that positive early results from an investigator-sponsored Phase 2 study of VOXZOGO® (vosoritide) in children with hypochondroplasia, will be presented at the 2024 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Toronto, Canada, March 12-16, 2024....
STOCKHOLM–– Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) today announced that its commercial partner Everest Medicines (HKEX 1952.HK) received approval from the Pharmaceutical Administration Bureau of the Macau Special Administrative Region, China. The approval for Nefecon is for the treatment of primary immunoglobulin A nephropathy (IgAN) in adults at risk...