Novartis' programme providing free access to its spinal muscular atrophy (SMA) gene therapy Zolgensma is being scaled back to a dozen countries worldwide, according to the company.
Astellas has been having some safety-related issues with its gene therapy programmes of late, but it shows no signs of lessened enthusiasm for the category – as a new alliance with US biote
Acadia Pharma's trofinetide has hit its efficacy targets in a phase 3 trial involving girls with the devastating neurological disorder Rett syndrome, although safety has dampened enthusiasm
Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRe
SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related ma
Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a
Sanofi is still plugging away with its oral BTK inhibitor tolebrutinib in multiple sclerosis, but has decided to abandon efforts to develop the drug for neuromuscular diso
In the newest episode of the pharmaphorum podcast, editor-in-chief Jonah Comstock speaks with Jameka Hill, senior director of clinical trial health equity at Moderna.