
University Start-Up Developing Inhaled Gene Therapies
A new company spun-off from a university lab consortium is advancing gene therapies delivered through the lungs to treat rare inherited respiratory diseases.
Science and EnterpriseData Support Placebo Group Alternatives in Rare Disease Trials
Findings from a review of medical #data and #records indicate matched #genetic control groups may not be needed in clinical trials testing treatments for Duchenne muscular dystrophy.
https://sciencebusiness.technewslit.com/?p=44426
#News #Science #Business #ClinicalTrials #DuchenneMuscularDystrophy #Neuroscience #Neurology #ControlGroup #Placebo #Statistics #RareDisease #InheritedDisease #RealWorldData

Data Support Placebo Group Alternatives in Rare Disease Trials
Findings from a review of medical data and records indicate matched genetic control groups may not be needed in clinical trials testing treatments for Duchenne muscular dystrophy.
Science and Enterprise