Topic: spinal muscular atrophy
Roche and partner PTC Therapeutics are the latest companies to show their hand in improving kids’ ability to move with spinal muscular atrophy.
The interim readout showed that two-fifths of infants who received PTC and Roche’s oral SMA drug were sitting eight months after starting treatment.
Blocking a pathway that causes healing cells to turn bad could offer a new way to treat neurological disorders.
Biogen has bolted on a couple of early-stage drug candidates for neuromuscular diseases via a $535 million deal with California startup AliveGen.
The Swiss pharma is walking away from olesoxime after running into “many difficulties in developing” the oral mitochondria-boosting drug.
This comes after two other biotechs had to pull their attempts last week.
Neuromuscular scores improved as expected in the first months of the trial, raising hopes the Novartis-bound gene therapy will deliver positive data.
Newly minted CEO Vas Narasimhan is paying a hefty premium to establish Novartis as a frontrunner in the emerging gene therapy sector.
The change sets the collaborators up to forge ahead with efforts to improve the sensitivity of spinal muscular atrophy screening tests.
AveXis is expanding trials of its gene therapy for children with spinal muscular atrophy (SMA) as it plays catch-up with Biogen and Ionis.