Remove tag targeted-solutions-chronic-diseases
article thumbnail

Realising milestones with gene therapy for SMA

European Pharmaceutical Review

We’ve achieved milestones no other gene therapy has accomplished to date by establishing flexible, tailored solutions enabling rapid access. Rare, genetic diseases like SMA are prime targets for gene therapy, but by their very nature, early signs can be difficult to recognise, delaying diagnosis and treatment.

article thumbnail

Don’t Wait—Uncover the Clinical Endpoints that Will Matter to Payers and HCPs

PM360

The shift toward longer, more adequate timeframes to establish proof of concept can be as long as three to four years, 1 given the complex requirements for developing and launching specialty drugs and cell and gene therapies (CGTs) for individuals with complex conditions or rare or orphan diseases.