Roche and PTC Therapeutics' Evrysdi for spinal muscular atrophy (SMA) has been approved by the FDA for younger children with the rare disease, extending its use to include infants less than
Roche’s Genentech unit has released further data from a key trial of Evrysdi, its oral treatment for spinal muscular atrophy (SMA), the rare muscle wasting disease that is often fatal in ba
The NHS should pay for Novartis’ £1.79 million one-off gene therapy for Spinal Muscular Atrophy (SMA) NICE has said, the most expensive treatment ever approved for funding.
Analysts have said the FDA’s request for a new trial will delay Novartis’ intrathecal formulation of its spinal muscular atrophy (SMA) gene therapy in older patients until 2023, potentially
The COVID-19 pandemic has bit into sales at Novartis, the big Swiss pharma said in second quarter results, after orders fell back following a period of stockpiling earlier in the year.
Endeavor BioMedicines has completed a third-round financing, raising an impressive $132.5 million to fund trials of its two clinical-stage drug candidates for pulmonary fi
It’s 2024, and in most clinical trials, data is still being moved from the EHR to the EDC manually – with employees reading data from one screen and typing it onto another.