Novartis releases long-term data for SMA gene therapy
Long-term follow-up study data for Zolgensma®, Novartis’ one-time gene therapy for spinal muscular atrophy (SMA), has shown promising overall milestone achievements.
List view / Grid view
Long-term follow-up study data for Zolgensma®, Novartis’ one-time gene therapy for spinal muscular atrophy (SMA), has shown promising overall milestone achievements.
Janssen’s single tablet of macitentan plus tadalafil significantly improved pulmonary hemodynamics in a Phase III trial for pulmonary arterial hypertension (PAH).
A National Institutes of Health (NIH)-supported trial has demonstrated the safety and efficiency of a freeze-dried thermostable tuberculosis vaccine.
Following finalisation a ten-year strategic partnership with the UK government, Moderna has chosen the location for its Innovation and Technology Centre (MITC).
The ABPI and Lilly have responded to the UK government’s confirmed plans to increase the payment percentage in the Statutory Scheme to control the costs of branded medicines.
The European Pharmacopoeia Commission’s new Excipients Strategy Working Party will address specificities of excipients in European Pharmacopoeia monographs.
Research has revealed efficiency of a multi-compartmental dissolution method used to predict in vivo performance of BCS Class IIa compounds.
In its latest meeting, the Committee for Medicinal Products for Human Use (CHMP) recommended eight new medicines for approval, including an enzyme replacement therapy for a rare disease.
In a Phase II trial, immunotherapy drug pembrolizumab significantly lowered the risk of recurrence for stage III-IV melanoma patients when given before and after surgery.
Bioconjugate-functionalised nanoparticles can isolate proteins from a bioreactor fast and inexpensively, MIT researchers have found.
CPI, AstraZeneca, Novartis, and The University of Manchester will collaborate to facilitate large-scale manufacture of oligonucleotides.
In a Phase III trial, a new cell therapy has shown potential for the first time to treat inflammation, a major contributor to heart failure.
A PEGylated enzyme replacement therapy for Fabry disease has received recommendation for a marketing authorisation from the European Medicines Agency (EMA)'s human medicines committee.
Between 2022 and 2035, the bioavailability enhancement services market will see an 11 percent compound annual growth rate, a report has predicted.
If approved, the cream formulation of the JAK inhibitor ruxolitinib will be the first treatment for repigmentation in non-segmental vitiligo available in the European Union.
US Pharmacopeia (USP) and American Type Culture Collection (ATCC) will collaborate to better support the biologics sector in R&D, process development and release of high-quality biologics.