Spinal Muscular Atrophies – 2

  • FIREFISH: A two part seamless multicenter study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in infants with spinal muscular atrophy type 1
  • SUNFISH: A two-part seamless, multi-center randomized, placebo-controlled, doubleblind study to investigate the safety, tolerabilty, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patients

 

 

Acronym

SUNFISH

FIREFISH

Study type
RBM RBM
Trial type International multicenter trial International multicenter trial
Intervention RO70344067 RO70344067
Principal investigator Odile Boespflug-Tanguy Odile Boespflug-Tanguy
Sponsor Roche Roche
Fundings Roche Roche
Study status Ongoing Ongoing
Recruitment status Ongoing Ongoing
Population Adult / Child Child
+ infos on clinicaltrials.gov + infos on clinicaltrials.gov