PTC Therapeutics (NJ, USA) has announced positive topline results from part II of the FIREFISH trial, demonstrating that risdiplam was well tolerated in infants with Type 1 spinal muscular atrophy.
Browsing: spinal muscular atrophy
A peek behind the paper – Melissa Bowerman on spinal muscular atrophy and the need for continued funding
In this Editorial, published in the journal Future Neurology, the author discusses the need for continued funding for spinal muscular atrophy.
New results from the NURTURE study have demonstrated that pre-symptomatic infants treated with Spinraza® achieved motor milestones not seen before.
This week we look at gammaCore™ for cluster headache, warnings of stroke risk with a multiple sclerosis drug and a gene therapy for spinal muscular atrophy.
NICE have issued a draft guidance against the use of Spinraza® (nusinersen) as an NHS treatment for spinal muscular atrophy.
This week’s industry news round-up reveals the latest updates around spinal muscular atrophy, Parkinson’s disease and acute stroke.
Giovanni Baranello speaks to Lauren Pulling about the FIREFISH trial for spinal muscular atrophy and what the next steps of the study include.
A preliminary study has revealed that an investigational drug may help increase protein levels in babies with spinal muscular atrophy.